Spinal Muscular Atrophy
Conditions
Keywords
SMA, SMN, SMNRx, ISIS-SMNRx, ISIS 396443, SHINE, IONIS-SMNRx, IONIS-SMN Rx, Spinraza, nusinersen
Brief summary
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.
Detailed description
This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, Biogen assumed responsibility for this study.
Interventions
DRUGnusinersen
Administered by intrathecal (IT) injection
Sponsors
Biogen
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Masking description
During the blinded loading period, the following participants will be masked: * Key site personnel (Investigator, Study Coordinator, and Outcomes Assessors) * Participant * The sponsor After the loading period has been completed, subsequent doses will be unblinded.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines. * Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks Key
Exclusion criteria
* Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study * Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study * Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study * Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer NOTE: Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to the end of the study (up to 2848 days) | AE:unfavorable and unintended sign, symptom, or disease temporally associated with study/use of an investigational drug, whether or not it's considered related to investigational drug. SAE:AE that in view of either Investigator/Sponsor, meets any of the following criteria: results in death;is life-threatening:i.e.poses risk of death, hospitalization/it's prolongation;results in a persistent or significant incapacity or substantial disruption of normal life functions;results in congenital anomaly or birth defect in offspring;is an important event in the opinion of Investigator/Sponsor. TEAE: if it was present prior to first dose of nusinersen or first sham procedure in index study and subsequently worsened in severity/was not present prior to first dose of nusinersen or first sham procedure in index study but subsequently appeared. |
| Number of Participants With Vital Sign Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | The vital sign assessments included blood pressure, temperature, pulse rate, and respiratory rate. Participants with abnormalities in these assessments recorded as AEs were reported. |
| Number of Participants With Weight Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Weight decrease was characterized by a decrease of \>=7% from baseline and weight increase was characterized by an increase of \>=7% from baseline. Participants with these abnormalities recorded as AEs were reported. |
| Number of Participants With Neurological Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Participants with abnormalities in neurological examinations recorded as AEs were reported. |
| Number of Participants With Laboratory Abnormalities Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Laboratory investigations included hematology, coagulation, serum chemistry and urinalysis parameters. Participants with abnormalities in these laboratory investigations recorded as AEs were reported. |
| Number of Participants With Coagulation Parameters Reported as AEs | From Day 1 up to the end of the study (up to 2848 days) | Coagulation parameters included activated partial thromboplastin time (aPTT) and international normalized ratio (INR). Participants with abnormalities in these coagulation parameters recorded as AEs were reported. |
| Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | From Day 1 up to the end of the study (up to 2848 days) | Clinical significance of abnormalities in 12 lead ECG was determined based on the investigator's discretion. |
| Number of Participants Taking Any Concomitant Medication | From Day 1 up to the end of the study (up to 2848 days) | A concomitant therapy is any non-protocol-specified drug or substance (including over-the-counter medications, herbal medications, and vitamin supplements) administered between the beginning of screening and the last telephone contact or study visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Not Requiring Permanent Ventilation | MMDR Period: Up to Day 1800 | Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event. |
| Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Baseline, Day 2198 | The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness, including infants with SMA. Participants who are ≥2 years will be continued to be assessed until a CHOP INTEND maximum score of 64 is achieved. It includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning. |
| Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | MMDR Period: Baseline, MMDR Day 1800 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. |
| Change From Baseline in Revised Upper Limb Module (RULM) Total Score | MMDR Period: Baseline, MMDR Day 1800 | RULM Test is used in participants with SMA to assess upper limb functional ability items and has total of 20 items with an entry item that serves as functional class identification and does not contribute to total score. Remaining 19 scorable items reflect different functional domains and graded on 3-point system with score of 0 (unable), 1 (able, with modification), and 2 (able, no difficulty). There is only 1 item that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for total score (0-37), higher scores indicating increased upper limb function. Positive change from baseline indicates improvement. |
| Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Baseline, Day 2670 | The 6MWT measures the distance an individual can walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes. |
| Number of Participants Who Experienced Contracture Assesment | MMDR Period: At MMDR Day 1800 | Contracture assessment is performed to assess the motor performance in SMA. The number of participants who experienced at least one contracture at any location and severe contractures in any of the five locations (hip flexors, knee flexors, ankle planter flexors, elbow flexors, forearm flexors) are reported in this outcome measure. |
| Change From Baseline in Compound Muscular Action Potential (CMAP) | MMDR Period: Baseline, MMDR Day 1800 | CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. Peroneal amplitude (PA) and ulnar amplitude (UA) data is reported in this OM. Score \<0 indicated worse response and \>0 indicated better response than the normal matched population. Score change \<0 indicated worsening and \>0 indicated improvement as compared to baseline. |
| Change From Baseline in Body Length | MMDR Period: up to Day 1800 | Participants were analyzed for change in growth parameter of body length to evaluate clinical efficacy. The body length was calculated using either WHO or Centers for Disease Control and Prevention (CDC) scales. The CDC scale allows to calculate the body length up to 20 years, while the WHO scale allows to calculate it only up to 10 years. |
| Total Number of Hospitalizations Due to Serious Adverse Events | Up to day 2520 | Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization. |
| Change From Baseline in Weight for Age Percentile | MMDR Period: up to Day 1800 | Participants who were below the age of 36 months were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. The weight for age percentile was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight for age percentile up to 20 years, while the WHO scale allows to calculate it only up to 10 years. |
| Percentage of CMAP Responders | MMDR Period: At Day 1800 | CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. A participant was defined as a responder if they had a peroneal amplitude ≥1 mV at last visit (including the amplitude ≥1 mV at baseline and also demonstrated as such at last visit). |
| Number of Participants Who Achieved Motor Milestones | MMDR Period: up to Day 1800 | Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support (SWS), standing with assistance (SWA), hands and knees crawling (HKC), and walking alone (WA). |
| Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | MMDR Period: At Day 1800 | Items on the PedsQL generic core scale are reverse scored and transformed to a 0-100 scale. The PedsQL parent (P) and self (S) reported questionnaire was collected for participants from 2 to 25 years of age. Four dimensions were collected: Physical, Emotional, Social and School functioning and each item was scored on a 5 point ordinal scale. 0 (never) =100, 1 (almost never) = 75, 2 (sometimes)= 50, 3 (often) = 25, 4 (almost always) = 0. A total score was calculated as the sum of all the items over the number of items answered on all the scales. If more than 50% of items or more were missing, the scale score was not computed. Higher scores indicated better health related quality of life. |
| Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | MMDR Period: up to Day 1800 | The ACEND is a questionnaire that includes a total of seven domains assessing physical impact (including feeding/grooming/dressing, sitting/play, transfers, and mobility) and general caregiver impact (including time, emotion, and finance) and each domain comprises several items. The total score (TS) for each domain will be calculated on a scale of 0 to 100. Higher scores indicate a greater impact on the caregiver. |
| Number of Participants With Disease-related Hospitalizations and AEs | MMDR Period: up to Day 1800 | — |
| Survival Rate | MMDR Period: up to Day 1800 | Survival rate was defined as the percentage of participants alive during the study and was estimated from the Kaplan Meier (KM) curve for time to death. |
| Change From Baseline in Weight | MMDR Period: up to Day 1800 | Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. The weight was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight up to 20 years, while the WHO scale allows to calculate it only up to 10 years. |
| Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | MMDR Period: At Day 1800 | The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone and walking alone. Mean of number of new milestones achieved was calculated and reported in this outcome measure. |
| Number of Participants Who Achieved Standing Alone and Walking With Assistance | MMDR Period: up to Day 1800 | Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: SWS, SWA, HKC, WWA, WA and standing alone (SA). SA and WWA were assessed in this outcome measure. |
| Total Number of Hospitalizations Due to Serious Respiratory Events | Up to day 2520 | Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization. |
| Percent of Time in Hospitalization | Upto day 2160 | — |
| Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | MMDR Period: Baseline, MMDR Day 1800 | Cobb angle is a measurement of the degree of side-to-side spinal curvature used to define scoliosis. |
| Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | At Day 309 | HINE is evaluated in infants between 2-24 months of age. It's a simple, standardized instrument including 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of HINE (HINE-2) was assessed, which evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform task and score of 2, 3, or 4 indicating full milestone development. Total score is calculated by summing item scores to give maximum possible score of 26. Higher score indicates good neurological function. |
| Number of Participants Who Died or Met Permanent Ventilation | MMDR Period: Up to Day 1800 | Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event. |
Countries
Australia, Belgium, Canada, France, Germany, Hong Kong, Italy, Japan, South Korea, Spain, Sweden, Turkey (Türkiye), United Kingdom, United States
Participant flow
Recruitment details
Participants were enrolled at the investigative sites in Australia, Belgium, Canada, Germany, Spain, France, United Kingdom, Hong Kong, Italy, Japan, Republic of Korea, Sweden, Turkey, and the United States from 04 November 2015 to 21 August 2023.
Pre-assignment details
A total of 292 participants with infantile and later onset spinal muscular atrophy (SMA) who previously participated in ISIS 396443-CS3B\[NCT02193074\], ISIS 396443-CS4\[NCT02292537\], ISIS 396443-CS3A\[NCT01839656\], ISIS 396443-CS12\[NCT02052791\] and 232SM202\[NCT02462759\] were enrolled and treated in this study.
Participants by arm
| Arm | Count |
|---|---|
| Infantile SMA Onset CS3A Participants who received nusinersen in study ISIS 396443-CS3A, in an open-label period, received maintenance doses of nusinersen, IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 13 |
| Infantile SMA Onset CS3B Previous Control Participants who received sham procedures in study ISIS 396443-CS3B, in a double-blind period, received 4 loading doses of nusinersen IT injection on Days 1, 15, 29, and 64 followed by maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 24 |
| Infantile SMA Onset CS3B Previous ISIS 396443 Participants who received nusinersen in study ISIS 396443-CS3B, in the double-blind period, received 3 sham procedures on Days 1, 15, and 64 and 1 loading dose of nusinersen IT injection on Day 29 followed by maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 65 |
| Infantile SMA Onset 232SM202 Participants who received nusinersen in study 232SM202 (i.e., Part 2) in an open-label period, received maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 12 |
| Later SMA Onset CS12 Type 2 Participants who received nusinersen in study ISIS 396443-CS12 in an open-label period, received maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 20 |
| Later SMA Onset CS12 Type 3 Participants who received nusinersen in study ISIS 396443-CS12 in an open-label period, received maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 25 |
| Later SMA Onset CS4 Previous Control Participants who received sham procedures in the study ISIS 396443-CS4 in a double-blind period received 3 loading doses of nusinersen administered by IT injection on Days 1, 29, and 85 followed by maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 42 |
| Later SMA Onset CS4 Previous ISIS 396443 Participants who received nusinersen in the study ISIS 396443-CS4 in a double-blind period received 2 loading doses of nusinersen administered by IT injection on Days 1 and 85 and 1 sham procedure on Day 29 followed by maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 83 |
| Later SMA Onset 232SM202 Participants who received nusinersen in study 232SM202 (i.e., Part 2) in an open-label period, received maintenance doses of nusinersen IT injection, on days 1, 120, 240, 360, 480, 600, 720, 840, 960, 1080, 1200, 1320, 1440, 1560, and 1680 in MMDR period of this study. | 8 |
| Total | 292 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 | FG007 | FG008 |
|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study | Adverse Event | 0 | 5 | 8 | 1 | 0 | 0 | 2 | 2 | 0 |
| Overall Study | Commercial drug | 0 | 1 | 6 | 3 | 0 | 0 | 7 | 11 | 2 |
| Overall Study | Physician Decision | 0 | 2 | 0 | 0 | 0 | 0 | 0 | 1 | 0 |
| Overall Study | Reason Not Specified | 1 | 3 | 4 | 0 | 0 | 1 | 1 | 6 | 1 |
| Overall Study | Withdrawal by Subject | 2 | 3 | 5 | 1 | 8 | 4 | 13 | 19 | 0 |
Baseline characteristics
| Characteristic | Total | Later SMA Onset 232SM202 | Later SMA Onset CS4 Previous ISIS 396443 | Later SMA Onset CS4 Previous Control | Later SMA Onset CS12 Type 3 | Later SMA Onset CS12 Type 2 | Infantile SMA Onset 232SM202 | Infantile SMA Onset CS3B Previous ISIS 396443 | Infantile SMA Onset CS3B Previous Control | Infantile SMA Onset CS3A |
|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized Adolescents (12-17 years) | 12 Participants | 0 Participants | 0 Participants | 0 Participants | 12 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Customized Adults (18-64 years) | 4 Participants | 0 Participants | 0 Participants | 0 Participants | 3 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Customized Children (2-11 years) | 187 Participants | 8 Participants | 83 Participants | 42 Participants | 10 Participants | 19 Participants | 12 Participants | 0 Participants | 0 Participants | 13 Participants |
| Age, Customized Infants and toddlers (28 days-23 months) | 89 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 65 Participants | 24 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 29 Participants | 1 Participants | 4 Participants | 0 Participants | 6 Participants | 2 Participants | 2 Participants | 10 Participants | 3 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 232 Participants | 5 Participants | 69 Participants | 37 Participants | 19 Participants | 18 Participants | 7 Participants | 47 Participants | 18 Participants | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 31 Participants | 2 Participants | 10 Participants | 5 Participants | 0 Participants | 0 Participants | 3 Participants | 8 Participants | 3 Participants | 0 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 2 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 33 Participants | 0 Participants | 16 Participants | 7 Participants | 1 Participants | 1 Participants | 4 Participants | 2 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Black or African American | 6 Participants | 0 Participants | 1 Participants | 1 Participants | 1 Participants | 0 Participants | 0 Participants | 3 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Multiple | 11 Participants | 0 Participants | 3 Participants | 3 Participants | 0 Participants | 1 Participants | 0 Participants | 1 Participants | 2 Participants | 1 Participants |
| Race/Ethnicity, Customized Not reported | 31 Participants | 2 Participants | 10 Participants | 5 Participants | 0 Participants | 0 Participants | 3 Participants | 8 Participants | 3 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 5 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 3 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized White | 204 Participants | 4 Participants | 53 Participants | 26 Participants | 23 Participants | 18 Participants | 5 Participants | 47 Participants | 18 Participants | 10 Participants |
| Sex: Female, Male Female | 156 Participants | 3 Participants | 46 Participants | 21 Participants | 15 Participants | 8 Participants | 6 Participants | 36 Participants | 15 Participants | 6 Participants |
| Sex: Female, Male Male | 136 Participants | 5 Participants | 37 Participants | 21 Participants | 10 Participants | 12 Participants | 6 Participants | 29 Participants | 9 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 5 / 24 | 8 / 65 | 1 / 12 | 0 / 8 | 0 / 20 | 0 / 25 | 1 / 42 | 1 / 83 |
| other Total, other adverse events | 13 / 13 | 23 / 24 | 65 / 65 | 12 / 12 | 8 / 8 | 20 / 20 | 25 / 25 | 40 / 42 | 79 / 83 |
| serious Total, serious adverse events | 11 / 13 | 23 / 24 | 59 / 65 | 9 / 12 | 5 / 8 | 12 / 20 | 6 / 25 | 26 / 42 | 47 / 83 |
Outcome results
Primary
Number of Participants Taking Any Concomitant Medication
A concomitant therapy is any non-protocol-specified drug or substance (including over-the-counter medications, herbal medications, and vitamin supplements) administered between the beginning of screening and the last telephone contact or study visit.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Taking Any Concomitant Medication | 13 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Taking Any Concomitant Medication | 24 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Taking Any Concomitant Medication | 65 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Taking Any Concomitant Medication | 12 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Taking Any Concomitant Medication | 20 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Taking Any Concomitant Medication | 25 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Taking Any Concomitant Medication | 42 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Taking Any Concomitant Medication | 83 Participants |
| Later SMA Onset 232SM202 | Number of Participants Taking Any Concomitant Medication | 8 Participants |
Primary
Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results
Clinical significance of abnormalities in 12 lead ECG was determined based on the investigator's discretion.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Number analyzed' signifies number of participants evaluable for this outcome measure.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 2 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 6 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Clinically Significant Shifts in12 Lead Electrocardiogram (ECG) Results | 0 Participants |
Primary
Number of Participants With Coagulation Parameters Reported as AEs
Coagulation parameters included activated partial thromboplastin time (aPTT) and international normalized ratio (INR). Participants with abnormalities in these coagulation parameters recorded as AEs were reported.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 2 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | Activated Partial Thromboplastin Time Prolonged | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | Coagulopathy | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Coagulation Parameters Reported as AEs | International Normalised Ratio Abnormal | 0 Participants |
Primary
Number of Participants With Laboratory Abnormalities Reported as AEs
Laboratory investigations included hematology, coagulation, serum chemistry and urinalysis parameters. Participants with abnormalities in these laboratory investigations recorded as AEs were reported.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 3 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 2 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 3 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 2 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 2 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 3 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 6 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 10 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 7 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 5 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 11 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 4 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 2 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 2 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 2 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 2 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 2 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 4 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 4 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 6 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 3 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 7 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 8 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypercalcaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Electrolyte Imbalance | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukopenia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Ketonuria | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pyuria | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine Positive | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoalbuminaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Alanine Aminotransferase Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bilirubinuria | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocytosis | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Crystal Urine Present | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cells Urine | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Aspartate Aminotransferase Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | White Blood Cell Count Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Leukocyturia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Albumin Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Liver Function Test Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haematuria | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Anaemia | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Mean Platelet Volume Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urine Ketone Body Present | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Bicarbonate Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophil Count Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Transaminases Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Calcium Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Red Blood Cell Count Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Haemoglobin Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Protein Urine Present | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Gamma-Glutamyltransferase Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Full Blood Count Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Osmolarity Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Bandaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Blood Potassium Abnormal | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Proteinuria | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypoglycaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Pancytopenia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyponatraemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypokalaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutropenia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hepatic Enzyme Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypochloraemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytopenia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Urinary Tract Infection | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypocalcaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Thrombocytosis | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Lymphopenia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Platelet Count Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypernatraemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hypertransaminasaemia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Neutrophilia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Laboratory Abnormalities Reported as AEs | Hyperglycaemia | 0 Participants |
Primary
Number of Participants With Neurological Abnormalities Reported as AEs
Participants with abnormalities in neurological examinations recorded as AEs were reported.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 2 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 4 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 2 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 3 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 4 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Tremor | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Areflexia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Hyporeflexia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Nystagmus | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Motor Dysfunction | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Muscle Contractions Involuntary | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Myoclonus | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Neurological Abnormalities Reported as AEs | Paraesthesia | 1 Participants |
Primary
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
AE:unfavorable and unintended sign, symptom, or disease temporally associated with study/use of an investigational drug, whether or not it's considered related to investigational drug. SAE:AE that in view of either Investigator/Sponsor, meets any of the following criteria: results in death;is life-threatening:i.e.poses risk of death, hospitalization/it's prolongation;results in a persistent or significant incapacity or substantial disruption of normal life functions;results in congenital anomaly or birth defect in offspring;is an important event in the opinion of Investigator/Sponsor. TEAE: if it was present prior to first dose of nusinersen or first sham procedure in index study and subsequently worsened in severity/was not present prior to first dose of nusinersen or first sham procedure in index study but subsequently appeared.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 13 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 11 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 24 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 23 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 65 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 59 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 12 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 9 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 20 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 12 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 6 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 25 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 26 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 42 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 80 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 47 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs | 8 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | SAEs | 5 Participants |
Primary
Number of Participants With Vital Sign Abnormalities Reported as AEs
The vital sign assessments included blood pressure, temperature, pulse rate, and respiratory rate. Participants with abnormalities in these assessments recorded as AEs were reported.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 2 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 13 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 3 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 9 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 2 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 15 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 4 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 5 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 6 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 4 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 50 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 20 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 2 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 5 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 3 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 10 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 3 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 2 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 21 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 2 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 3 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 39 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Oxygen Saturation Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Body Temperature Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Bradycardia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachycardia | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Pyrexia | 5 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Heart Rate Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Respiratory Rate Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Vital Sign Abnormalities Reported as AEs | Tachypnoea | 0 Participants |
Primary
Number of Participants With Weight Abnormalities Reported as AEs
Weight decrease was characterized by a decrease of \>=7% from baseline and weight increase was characterized by an increase of \>=7% from baseline. Participants with these abnormalities recorded as AEs were reported.
Time frame: From Day 1 up to the end of the study (up to 2848 days)
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. '
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 1 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 5 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 1 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 3 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Decreased | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Weight Abnormalities Reported as AEs | Weight Increased | 0 Participants |
Secondary
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score
The ACEND is a questionnaire that includes a total of seven domains assessing physical impact (including feeding/grooming/dressing, sitting/play, transfers, and mobility) and general caregiver impact (including time, emotion, and finance) and each domain comprises several items. The total score (TS) for each domain will be calculated on a scale of 0 to 100. Higher scores indicate a greater impact on the caregiver.
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | -2.3 score on scale | Standard Deviation 14.93 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | 0.4 score on scale | Standard Deviation 7.24 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 0.8 score on scale | Standard Deviation 10.29 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | 5.0 score on scale | Standard Deviation 14.8 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | 4.4 score on scale | Standard Deviation 19.78 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | -1.0 score on scale | Standard Deviation 17.29 |
| Infantile SMA Onset CS3A | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 14.2 score on scale | Standard Deviation 21.56 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | 0.5 score on scale | Standard Deviation 1.72 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 0.6 score on scale | Standard Deviation 2.01 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | 6.8 score on scale | Standard Deviation 24.76 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | -0.4 score on scale | Standard Deviation 14.91 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | 0.7 score on scale | Standard Deviation 1.62 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | 6.3 score on scale | Standard Deviation 15.82 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | 7.3 score on scale | Standard Deviation 20.66 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | 7.5 score on scale | Standard Deviation 22.52 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | -2.1 score on scale | Standard Deviation 20.98 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | 0.0 score on scale | Standard Deviation 12.32 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | -0.7 score on scale | Standard Deviation 22.6 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | -4.1 score on scale | Standard Deviation 22.17 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 5.5 score on scale | Standard Deviation 28.01 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 18.5 score on scale | Standard Deviation 22.07 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 1.5 score on scale | Standard Deviation 22.21 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | -6.3 score on scale | Standard Deviation 21.65 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 9.2 score on scale | Standard Deviation 15.91 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | 4.4 score on scale | Standard Deviation 18.98 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | -5.5 score on scale | Standard Deviation 6.68 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | 2.1 score on scale | Standard Deviation 14.22 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | -12.1 score on scale | Standard Deviation 18.69 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | -2.4 score on scale | Standard Deviation 21 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | -13.0 score on scale | Standard Deviation 13.83 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | -0.8 score on scale | Standard Deviation 20.7 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 2.8 score on scale | Standard Deviation 12.04 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | -2.4 score on scale | Standard Deviation 18.14 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 2.1 score on scale | Standard Deviation 14.82 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | -5.8 score on scale | Standard Deviation 15.14 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | 1.4 score on scale | Standard Deviation 9.48 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | 4.1 score on scale | Standard Deviation 14.92 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 0.0 score on scale | Standard Deviation 0 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | -2.2 score on scale | Standard Deviation 7.86 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | 2.6 score on scale | Standard Deviation 18.36 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | 1.0 score on scale | Standard Deviation 13.25 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 0.6 score on scale | Standard Deviation 2.43 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | 1.4 score on scale | Standard Deviation 17.22 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | 1.9 score on scale | Standard Deviation 18.47 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | 4.7 score on scale | Standard Deviation 12.73 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | -3.6 score on scale | Standard Deviation 16.17 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | 7.2 score on scale | Standard Deviation 17.92 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | -1.8 score on scale | Standard Deviation 16.48 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 4.6 score on scale | Standard Deviation 18.9 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 5.9 score on scale | Standard Deviation 12.07 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | 0.5 score on scale | Standard Deviation 16.78 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | 1.7 score on scale | Standard Deviation 16.08 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | -2.5 score on scale | Standard Deviation 12.7 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | 2.1 score on scale | Standard Deviation 19.94 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | -5.8 score on scale | Standard Deviation 17.5 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | 1.2 score on scale | Standard Deviation 18.16 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Transfers TS: Change at MMDR Day 1800 | -4.0 score on scale | Standard Deviation 11.03 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Mobility TS: Change at MMDR Day 1800 | 6.7 score on scale | Standard Deviation 20.66 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Emotion TS: Change at MMDR Day 1800 | -18.1 score on scale | Standard Deviation 12.01 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Finance TS: Change at MMDR Day 1800 | -3.3 score on scale | Standard Deviation 8.16 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Feeding/Grooming/Dressing TS: Change MMDR Day1800 | 2.8 score on scale | Standard Deviation 7.72 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Time TS: Change at MMDR Day 1800 | -6.3 score on scale | Standard Deviation 14.25 |
| Later SMA Onset 232SM202 | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | Sitting/Play TS: Change at MMDR Day 1800 | -2.0 score on scale | Standard Deviation 4.9 |
Secondary
Change From Baseline in Body Length
Participants were analyzed for change in growth parameter of body length to evaluate clinical efficacy. The body length was calculated using either WHO or Centers for Disease Control and Prevention (CDC) scales. The CDC scale allows to calculate the body length up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Body Length | 24.2 centimeters (cm) | Standard Deviation 2.86 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Body Length | 19.9 centimeters (cm) | Standard Deviation 6.99 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Body Length | 29.4 centimeters (cm) | Standard Deviation 6.38 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Body Length | 18.8 centimeters (cm) | Standard Deviation 10.92 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Body Length | 18.8 centimeters (cm) | Standard Deviation 8.54 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Body Length | 23.1 centimeters (cm) | Standard Deviation 0 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Body Length | 23.7 centimeters (cm) | Standard Deviation 7.06 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Body Length | 24.6 centimeters (cm) | Standard Deviation 10.6 |
| Later SMA Onset 232SM202 | Change From Baseline in Body Length | -10.3 centimeters (cm) | Standard Deviation 57.63 |
Secondary
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale
The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness, including infants with SMA. Participants who are ≥2 years will be continued to be assessed until a CHOP INTEND maximum score of 64 is achieved. It includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning.
Time frame: Baseline, Day 2198
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for OM analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified time point. Only CS3A and CS3B arm groups were planned to be analyzed for this OM.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Baseline | 47.4 score on scale | Standard Deviation 13.09 |
| Infantile SMA Onset CS3A | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Change at Day 2198 | -6.0 score on scale | — |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Baseline | 17.3 score on scale | Standard Deviation 9.71 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Change at Day 2198 | 11.5 score on scale | Standard Deviation 12.21 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Baseline | 38.8 score on scale | Standard Deviation 9.43 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Change at Day 2198 | 4.7 score on scale | Standard Deviation 14.18 |
Secondary
Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit
Cobb angle is a measurement of the degree of side-to-side spinal curvature used to define scoliosis.
Time frame: MMDR Period: Baseline, MMDR Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 29.8 degree | Standard Deviation 9.46 |
| Infantile SMA Onset CS3A | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 22.7 degree | Standard Deviation 17.86 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 16.6 degree | Standard Deviation 13.38 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 16.9 degree | Standard Deviation 27.27 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 13.0 degree | Standard Deviation 29.91 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 34.8 degree | Standard Deviation 18.43 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 49.2 degree | Standard Deviation 13.19 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | -11.6 degree | Standard Deviation 24.65 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 38.5 degree | Standard Deviation 27.36 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | -3.8 degree | Standard Deviation 17.01 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 4.0 degree | Standard Deviation 7.78 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 29.8 degree | Standard Deviation 25.09 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 24.3 degree | Standard Deviation 19.08 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 19.8 degree | Standard Deviation 30.83 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | 18.8 degree | Standard Deviation 30.3 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 26.8 degree | Standard Deviation 20.33 |
| Later SMA Onset 232SM202 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Change at MMDR day 1800 | -13.8 degree | Standard Deviation 0 |
| Later SMA Onset 232SM202 | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | Baseline | 33.0 degree | Standard Deviation 16.94 |
Secondary
Change From Baseline in Compound Muscular Action Potential (CMAP)
CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. Peroneal amplitude (PA) and ulnar amplitude (UA) data is reported in this OM. Score \<0 indicated worse response and \>0 indicated better response than the normal matched population. Score change \<0 indicated worsening and \>0 indicated improvement as compared to baseline.
Time frame: MMDR Period: Baseline, MMDR Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.45 millivolt (mV) | Standard Deviation 0.837 |
| Infantile SMA Onset CS3A | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 3.02 millivolt (mV) | Standard Deviation 2.207 |
| Infantile SMA Onset CS3A | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | -0.41 millivolt (mV) | Standard Deviation 1.29 |
| Infantile SMA Onset CS3A | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 1.52 millivolt (mV) | Standard Deviation 1.563 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | 0.10 millivolt (mV) | Standard Deviation 0.243 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 0.35 millivolt (mV) | Standard Deviation 0.517 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.20 millivolt (mV) | Standard Deviation 0.188 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 0.20 millivolt (mV) | Standard Deviation 0.205 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 1.91 millivolt (mV) | Standard Deviation 1.367 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.67 millivolt (mV) | Standard Deviation 1.261 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 0.85 millivolt (mV) | Standard Deviation 0.944 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | 0.30 millivolt (mV) | Standard Deviation 1.395 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 0.99 millivolt (mV) | Standard Deviation 0.861 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 0.94 millivolt (mV) | Standard Deviation 0.929 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | 0.92 millivolt (mV) | Standard Deviation 1.901 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.15 millivolt (mV) | Standard Deviation 0.436 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | -0.59 millivolt (mV) | Standard Deviation 1.069 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 2.81 millivolt (mV) | Standard Deviation 1.723 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.04 millivolt (mV) | Standard Deviation 0.957 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 1.89 millivolt (mV) | Standard Deviation 1.369 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 6.76 millivolt (mV) | Standard Deviation 2.448 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.04 millivolt (mV) | Standard Deviation 1.641 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 2.71 millivolt (mV) | Standard Deviation 1.318 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | -0.08 millivolt (mV) | Standard Deviation 1.799 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 1.69 millivolt (mV) | Standard Deviation 1.133 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.32 millivolt (mV) | Standard Deviation 1.127 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 2.00 millivolt (mV) | Standard Deviation 2.189 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | 0.22 millivolt (mV) | Standard Deviation 4.832 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 2.71 millivolt (mV) | Standard Deviation 2.161 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | -0.28 millivolt (mV) | Standard Deviation 0.924 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 1.97 millivolt (mV) | Standard Deviation 1.586 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 0.52 millivolt (mV) | Standard Deviation 1.6 |
| Later SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Baseline | 1.35 millivolt (mV) | Standard Deviation 0.948 |
| Later SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | PA: Change at MMDR Day 1800 | 0.10 millivolt (mV) | Standard Deviation 1.414 |
| Later SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Change at MMDR Day 1800 | 2.77 millivolt (mV) | Standard Deviation 2.729 |
| Later SMA Onset 232SM202 | Change From Baseline in Compound Muscular Action Potential (CMAP) | UA: Baseline | 1.25 millivolt (mV) | Standard Deviation 0.935 |
Secondary
Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function.
Time frame: MMDR Period: Baseline, MMDR Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 14.5 score on scale | Standard Deviation 13.75 |
| Infantile SMA Onset CS3A | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | 3.4 score on scale | Standard Deviation 5.93 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 0.0 score on scale | Standard Deviation 0 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | 0.4 score on scale | Standard Deviation 0.88 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 7.3 score on scale | Standard Deviation 6.88 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | 6.0 score on scale | Standard Deviation 9.12 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 6.8 score on scale | Standard Deviation 6.06 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -2.3 score on scale | Standard Deviation 3.37 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 25.6 score on scale | Standard Deviation 14.23 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -7.0 score on scale | Standard Deviation 6.03 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -2.4 score on scale | Standard Deviation 3.41 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 53.6 score on scale | Standard Deviation 8.24 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -4.7 score on scale | Standard Deviation 7.2 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 21.1 score on scale | Standard Deviation 7.75 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 26.1 score on scale | Standard Deviation 10.99 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -6.2 score on scale | Standard Deviation 6.36 |
| Later SMA Onset 232SM202 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Baseline | 24.5 score on scale | Standard Deviation 12.64 |
| Later SMA Onset 232SM202 | Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Change at MMDR Day 1800 | -1.2 score on scale | Standard Deviation 9.83 |
Secondary
Change From Baseline in Revised Upper Limb Module (RULM) Total Score
RULM Test is used in participants with SMA to assess upper limb functional ability items and has total of 20 items with an entry item that serves as functional class identification and does not contribute to total score. Remaining 19 scorable items reflect different functional domains and graded on 3-point system with score of 0 (unable), 1 (able, with modification), and 2 (able, no difficulty). There is only 1 item that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for total score (0-37), higher scores indicating increased upper limb function. Positive change from baseline indicates improvement.
Time frame: MMDR Period: Baseline, MMDR Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 9.1 score on scale | Standard Deviation 4.7 |
| Infantile SMA Onset CS3A | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 11.8 score on scale | Standard Deviation 7.24 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 0.7 score on scale | Standard Deviation 0.58 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 1.7 score on scale | Standard Deviation 1.63 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 10.3 score on scale | Standard Deviation 6.05 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 10.0 score on scale | Standard Deviation 5.38 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 7.6 score on scale | Standard Deviation 6.23 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 3.3 score on scale | Standard Deviation 5.01 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 24.1 score on scale | Standard Deviation 6.14 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 0.4 score on scale | Standard Deviation 3.23 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 0.9 score on scale | Standard Deviation 1.76 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 35.9 score on scale | Standard Deviation 1.91 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 21.1 score on scale | Standard Deviation 4.26 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 2.4 score on scale | Standard Deviation 3.97 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 23.9 score on scale | Standard Deviation 5.69 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 1.2 score on scale | Standard Deviation 3.72 |
| Later SMA Onset 232SM202 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Change at MMDR Day 1800 | 3.2 score on scale | Standard Deviation 2.71 |
| Later SMA Onset 232SM202 | Change From Baseline in Revised Upper Limb Module (RULM) Total Score | Baseline | 21.4 score on scale | Standard Deviation 8.6 |
Secondary
Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT)
The 6MWT measures the distance an individual can walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes.
Time frame: Baseline, Day 2670
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint. Only CS12 Type 2 and CS12 Type 3 arm groups were planned to be analysed for this end point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Baseline | 0 meters | — |
| Infantile SMA Onset CS3A | Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Change at Day 2670 | 156.5 meters | — |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Baseline | 253.3 meters | Standard Deviation 182.74 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Total Distance Walked Over Time as Assessed by 6-Minute Walk Test (6MWT) | Change at Day 2670 | 91.5 meters | Standard Deviation 49.07 |
Secondary
Change From Baseline in Weight
Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. The weight was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Weight | 9.8 kilograms (kg) | Standard Deviation 3.48 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Weight | 7.7 kilograms (kg) | Standard Deviation 4.39 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Weight | 9.3 kilograms (kg) | Standard Deviation 3.25 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Weight | 9.5 kilograms (kg) | Standard Deviation 7.04 |
| Later SMA Onset CS12 Type 2 | Change From Baseline in Weight | 15.1 kilograms (kg) | Standard Deviation 5.96 |
| Later SMA Onset CS12 Type 3 | Change From Baseline in Weight | 13.8 kilograms (kg) | Standard Deviation 14.88 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Weight | 15.4 kilograms (kg) | Standard Deviation 8.05 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Weight | 15.2 kilograms (kg) | Standard Deviation 7.38 |
| Later SMA Onset 232SM202 | Change From Baseline in Weight | 11.7 kilograms (kg) | Standard Deviation 8.09 |
Secondary
Change From Baseline in Weight for Age Percentile
Participants who were below the age of 36 months were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. The weight for age percentile was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight for age percentile up to 20 years, while the WHO scale allows to calculate it only up to 10 years.
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Infantile SMA Onset CS3A | Change From Baseline in Weight for Age Percentile | 5.7 percentile | Standard Deviation 27.3 |
| Infantile SMA Onset CS3B Previous Control | Change From Baseline in Weight for Age Percentile | -5.3 percentile | Standard Deviation 28.97 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Change From Baseline in Weight for Age Percentile | 0.3 percentile | Standard Deviation 28.55 |
| Infantile SMA Onset 232SM202 | Change From Baseline in Weight for Age Percentile | -0.7 percentile | Standard Deviation 25.32 |
| Later SMA Onset CS4 Previous Control | Change From Baseline in Weight for Age Percentile | 0.9 percentile | Standard Deviation 17.22 |
| Later SMA Onset CS4 Previous ISIS 396443 | Change From Baseline in Weight for Age Percentile | 20.2 percentile | Standard Deviation 28.15 |
| Later SMA Onset 232SM202 | Change From Baseline in Weight for Age Percentile | 4.0 percentile | Standard Deviation 33.11 |
Secondary
Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria
The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone and walking alone. Mean of number of new milestones achieved was calculated and reported in this outcome measure.
Time frame: MMDR Period: At Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Infantile SMA Onset CS3A | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | 0.4 motor milestone | Standard Deviation 0.73 |
| Infantile SMA Onset CS3B Previous Control | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | 0.0 motor milestone | Standard Deviation 0 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | 0.7 motor milestone | Standard Deviation 1.15 |
| Infantile SMA Onset 232SM202 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | 0.0 motor milestone | Standard Deviation 0.53 |
| Later SMA Onset CS12 Type 2 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | -0.6 motor milestone | Standard Deviation 0.79 |
| Later SMA Onset CS12 Type 3 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | -0.1 motor milestone | Standard Deviation 0.73 |
| Later SMA Onset CS4 Previous Control | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | -0.1 motor milestone | Standard Deviation 0.32 |
| Later SMA Onset CS4 Previous ISIS 396443 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | -0.2 motor milestone | Standard Deviation 0.6 |
| Later SMA Onset 232SM202 | Mean Number of New Motor Milestones Achieved as Assessed by World Health Organization (WHO) Criteria | -0.2 motor milestone | Standard Deviation 0.75 |
Secondary
Number of Participants Not Requiring Permanent Ventilation
Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event.
Time frame: MMDR Period: Up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Not Requiring Permanent Ventilation | 13 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Not Requiring Permanent Ventilation | 20 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Not Requiring Permanent Ventilation | 61 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Not Requiring Permanent Ventilation | 11 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Not Requiring Permanent Ventilation | 20 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Not Requiring Permanent Ventilation | 42 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Not Requiring Permanent Ventilation | 83 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Not Requiring Permanent Ventilation | 25 Participants |
| Later SMA Onset 232SM202 | Number of Participants Not Requiring Permanent Ventilation | 8 Participants |
Secondary
Number of Participants Who Achieved Motor Milestones
Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support (SWS), standing with assistance (SWA), hands and knees crawling (HKC), and walking alone (WA).
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 3 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 5 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 16 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 11 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 4 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 3 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 13 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 4 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 23 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 20 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 16 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 1 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 20 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 3 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 3 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 27 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 3 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 18 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 58 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 2 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | WA: Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | HKC: Inability at Baseline, Achieved at Last Visit | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWA:Achieved at Baseline&Maintained to Last Visit | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWS:Inability at Baseline, Achieved at Last visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWA:Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | WA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | SWS:Achieved at Baseline&Maintained to Last Visit | 5 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Motor Milestones | HKC:Achieved at Baseline&Maintained to Last Visit | 2 Participants |
Secondary
Number of Participants Who Achieved Standing Alone and Walking With Assistance
Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: SWS, SWA, HKC, WWA, WA and standing alone (SA). SA and WWA were assessed in this outcome measure.
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 2 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 2 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 0 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 19 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 19 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 2 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 2 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Inability at Baseline, Achieved at Last Visit | 0 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | SA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Achieved Standing Alone and Walking With Assistance | WWA: Achieved at Baseline & Maintained to Last Visit | 1 Participants |
Secondary
Number of Participants Who Died or Met Permanent Ventilation
Permanent ventilation was defined as tracheostomy or \>=16 hours of ventilator support per day continuously for \>21 days in the absence of an acute reversible event.
Time frame: MMDR Period: Up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Who Died or Met Permanent Ventilation | 0 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Died or Met Permanent Ventilation | 6 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Died or Met Permanent Ventilation | 11 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Died or Met Permanent Ventilation | 1 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Died or Met Permanent Ventilation | 0 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Died or Met Permanent Ventilation | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Died or Met Permanent Ventilation | 0 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Died or Met Permanent Ventilation | 2 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Died or Met Permanent Ventilation | 0 Participants |
Secondary
Number of Participants Who Experienced Contracture Assesment
Contracture assessment is performed to assess the motor performance in SMA. The number of participants who experienced at least one contracture at any location and severe contractures in any of the five locations (hip flexors, knee flexors, ankle planter flexors, elbow flexors, forearm flexors) are reported in this outcome measure.
Time frame: MMDR Period: At MMDR Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 9 Participants |
| Infantile SMA Onset CS3A | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 3 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 10 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 3 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 36 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 16 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 4 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 8 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 13 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 6 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 8 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 1 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 9 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 19 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 16 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 44 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Experienced Contracture Assesment | At least one contracture at any location | 5 Participants |
| Later SMA Onset 232SM202 | Number of Participants Who Experienced Contracture Assesment | Severe contractures in any of the five locations | 2 Participants |
Secondary
Number of Participants With Disease-related Hospitalizations and AEs
Time frame: MMDR Period: up to Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 6 Participants |
| Infantile SMA Onset CS3A | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 11 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 9 Participants |
| Infantile SMA Onset CS3B Previous Control | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 21 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 27 Participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 60 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 5 Participants |
| Infantile SMA Onset 232SM202 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 7 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 9 Participants |
| Later SMA Onset CS12 Type 2 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 16 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 12 Participants |
| Later SMA Onset CS12 Type 3 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 0 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 36 Participants |
| Later SMA Onset CS4 Previous Control | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 13 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 23 Participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 68 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related hospitalization | 3 Participants |
| Later SMA Onset 232SM202 | Number of Participants With Disease-related Hospitalizations and AEs | Disease-related AEs | 8 Participants |
Secondary
Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain
Items on the PedsQL generic core scale are reverse scored and transformed to a 0-100 scale. The PedsQL parent (P) and self (S) reported questionnaire was collected for participants from 2 to 25 years of age. Four dimensions were collected: Physical, Emotional, Social and School functioning and each item was scored on a 5 point ordinal scale. 0 (never) =100, 1 (almost never) = 75, 2 (sometimes)= 50, 3 (often) = 25, 4 (almost always) = 0. A total score was calculated as the sum of all the items over the number of items answered on all the scales. If more than 50% of items or more were missing, the scale score was not computed. Higher scores indicated better health related quality of life.
Time frame: MMDR Period: At Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 25.0 score on scale | Standard Deviation 27.55 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 48.8 score on scale | Standard Deviation 12.17 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 47.5 score on scale | Standard Deviation 12.82 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 67.5 score on scale | Standard Deviation 22.83 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 54.4 score on scale | Standard Deviation 14.25 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 61.9 score on scale | Standard Deviation 12.52 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 68.1 score on scale | Standard Deviation 13.08 |
| Infantile SMA Onset CS3A | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 37.1 score on scale | Standard Deviation 11.26 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 40.8 score on scale | Standard Deviation 33.23 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 22.4 score on scale | Standard Deviation 31.52 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 61.7 score on scale | Standard Deviation 26.39 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 54.0 score on scale | Standard Deviation 23.66 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 50.8 score on scale | Standard Deviation 41.52 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 69.5 score on scale | Standard Deviation 17.23 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 19.1 score on scale | Standard Deviation 24.54 |
| Infantile SMA Onset CS3B Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 48.5 score on scale | Standard Deviation 31.23 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 24.9 score on scale | Standard Deviation 22.77 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 51.3 score on scale | Standard Deviation 20.06 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 67.1 score on scale | Standard Deviation 22.87 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 51.4 score on scale | Standard Deviation 21.36 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 58.7 score on scale | Standard Deviation 14.9 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 66.1 score on scale | Standard Deviation 17.66 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 47.5 score on scale | Standard Deviation 25.31 |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 63.3 score on scale | Standard Deviation 20.71 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 64.3 score on scale | Standard Deviation 22.63 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 67.1 score on scale | Standard Deviation 19.55 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 75.7 score on scale | Standard Deviation 20.09 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 63.6 score on scale | Standard Deviation 18.64 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 65.0 score on scale | Standard Deviation 26.46 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 32.6 score on scale | Standard Deviation 30.07 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 71.4 score on scale | Standard Deviation 14.64 |
| Infantile SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 32.6 score on scale | Standard Deviation 20.48 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 77.3 score on scale | Standard Deviation 13.33 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 71.9 score on scale | Standard Deviation 24.46 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 77.7 score on scale | Standard Deviation 11.66 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 72.7 score on scale | Standard Deviation 19.75 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 37.5 score on scale | Standard Deviation 13.68 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 79.2 score on scale | Standard Deviation 14.41 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 76.5 score on scale | Standard Deviation 13.45 |
| Later SMA Onset CS12 Type 2 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 28.6 score on scale | Standard Deviation 14.31 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 46.1 score on scale | Standard Deviation 21.47 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 52.7 score on scale | Standard Deviation 16.94 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 78.0 score on scale | Standard Deviation 21.24 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 80.8 score on scale | Standard Deviation 13.11 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 77.8 score on scale | Standard Deviation 15.09 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 71.8 score on scale | Standard Deviation 19.84 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 72.1 score on scale | Standard Deviation 16.96 |
| Later SMA Onset CS12 Type 3 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 78.5 score on scale | Standard Deviation 18.44 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 70.0 score on scale | Standard Deviation 20.65 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 45.4 score on scale | Standard Deviation 13.85 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 84.6 score on scale | Standard Deviation 15.42 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 81.8 score on scale | Standard Deviation 14.89 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 83.3 score on scale | Standard Deviation 15.07 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 78.8 score on scale | Standard Deviation 20.19 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 78.0 score on scale | Standard Deviation 18.74 |
| Later SMA Onset CS4 Previous Control | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 35.2 score on scale | Standard Deviation 18.19 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 77.1 score on scale | Standard Deviation 15.22 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 76.3 score on scale | Standard Deviation 13.88 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 74.5 score on scale | Standard Deviation 22.8 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 78.2 score on scale | Standard Deviation 19.6 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 78.0 score on scale | Standard Deviation 14.75 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 71.0 score on scale | Standard Deviation 17.5 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 39.7 score on scale | Standard Deviation 17.97 |
| Later SMA Onset CS4 Previous ISIS 396443 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 33.5 score on scale | Standard Deviation 18.41 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Social Functioning Report | 60.8 score on scale | Standard Deviation 13.2 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Emotional Functioning Report | 74.2 score on scale | Standard Deviation 11.14 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: Physical Functioning Report | 20.8 score on scale | Standard Deviation 5.82 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Physical Functioning Report | 53.7 score on scale | Standard Deviation 24.46 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Social Functioning | 80.0 score on scale | Standard Deviation 12.25 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | P: School/Work Functioning Report | 79.2 score on scale | Standard Deviation 9.7 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: Emotional Functioning Report | 67.0 score on scale | Standard Deviation 16.43 |
| Later SMA Onset 232SM202 | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | S: School/Work Functioning Report | 82.0 score on scale | Standard Deviation 11.51 |
Secondary
Percentage of CMAP Responders
CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. A participant was defined as a responder if they had a peroneal amplitude ≥1 mV at last visit (including the amplitude ≥1 mV at baseline and also demonstrated as such at last visit).
Time frame: MMDR Period: At Day 1800
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Infantile SMA Onset CS3A | Percentage of CMAP Responders | 85 percentage of responders |
| Infantile SMA Onset CS3B Previous Control | Percentage of CMAP Responders | 0 percentage of responders |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of CMAP Responders | 72 percentage of responders |
| Infantile SMA Onset 232SM202 | Percentage of CMAP Responders | 33 percentage of responders |
| Later SMA Onset CS12 Type 2 | Percentage of CMAP Responders | 65 percentage of responders |
| Later SMA Onset CS12 Type 3 | Percentage of CMAP Responders | 67 percentage of responders |
| Later SMA Onset CS4 Previous Control | Percentage of CMAP Responders | 60 percentage of responders |
| Later SMA Onset CS4 Previous ISIS 396443 | Percentage of CMAP Responders | 51 percentage of responders |
| Later SMA Onset 232SM202 | Percentage of CMAP Responders | 25 percentage of responders |
Secondary
Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE)
HINE is evaluated in infants between 2-24 months of age. It's a simple, standardized instrument including 26 items assessing different aspects of neurological examinations, such as cranial nerves, posture, movements, tone, and reflexes. In this study, Module 2 of HINE (HINE-2) was assessed, which evaluates 8 developmental milestones (head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking) scored on a 3, 4, or 5-point scale, with 0 indicating inability to perform task and score of 2, 3, or 4 indicating full milestone development. Total score is calculated by summing item scores to give maximum possible score of 26. Higher score indicates good neurological function.
Time frame: At Day 309
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. Only CS3A and CS3B arm groups were planned to be analyzed for this outcome measure (OM).
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sits With Support at Hips | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Unable to Maintain Head Upright | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Wobbles | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: All the Time Maintained Upright | 75 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Cannot Sit | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Props | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Stable sit | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Pivots (rotates) | 63 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: No kicking | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Kick horizontally legs do not lift | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Upward (vertically) | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches leg | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches toes | 88 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: No Rolling | 25 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Rolling to Side | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Prone to Supine | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Supine to Prone | 63 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Does not Lift Head | 63 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Elbow | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Outstretched Hand | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling Flat on Abdomen | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling on Hands and Knees | 25 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Does not support weight | 38 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Supports weight | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands with support | 25 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands unaided | 25 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: No Walking | 75 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Bouncing | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Cruising (Walks Holding on) | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Walking Independently | 13 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:No Grasp | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Uses Whole Hand | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Index Finger&Thumb;Immature Grasp | 0 percentage of participants |
| Infantile SMA Onset CS3A | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Pincer Grasp | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: No Walking | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches toes | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: No Rolling | 91 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Bouncing | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Rolling to Side | 9 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Prone to Supine | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Pincer Grasp | 18 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Supine to Prone | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Cruising (Walks Holding on) | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Does not Lift Head | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Elbow | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Index Finger&Thumb;Immature Grasp | 14 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Outstretched Hand | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Walking Independently | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling Flat on Abdomen | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling on Hands and Knees | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Does not support weight | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:No Grasp | 14 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Unable to Maintain Head Upright | 86 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Supports weight | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Wobbles | 9 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: All the Time Maintained Upright | 5 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Cannot Sit | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sits With Support at Hips | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands with support | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Props | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Stable sit | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Pivots (rotates) | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands unaided | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: No kicking | 68 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Kick horizontally legs do not lift | 32 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Uses Whole Hand | 55 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Upward (vertically) | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches leg | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Does not support weight | 79 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches leg | 11 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: No Walking | 95 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Props | 8 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Touches toes | 32 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Uses Whole Hand | 18 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands with support | 10 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: No Rolling | 27 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Unable to Maintain Head Upright | 34 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: No kicking | 18 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Rolling to Side | 37 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Bouncing | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Stable sit | 19 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Prone to Supine | 5 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: Wobbles | 18 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Supports weight | 11 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Rolling: Supine to Prone | 31 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Pincer Grasp | 52 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Head Control: All the Time Maintained Upright | 48 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Does not Lift Head | 81 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Cruising (Walks Holding on) | 5 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:No Grasp | 6 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Elbow | 13 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Standing: Stands unaided | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Cannot Sit | 39 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: On Outstretched Hand | 2 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sitting: Pivots (rotates) | 15 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Sits With Support at Hips | 19 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling Flat on Abdomen | 3 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Walking: Walking Independently | 0 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Upward (vertically) | 10 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Crawling: Crawling on Hands and Knees | 2 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Voluntary Grasp:Index Finger&Thumb;Immature Grasp | 21 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percentage of Participants With <2 Years of Age Who Attained Motor Milestones as Assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) | Ability to Kick: Kick horizontally legs do not lift | 29 percentage of participants |
Secondary
Percent of Time in Hospitalization
Time frame: Upto day 2160
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 1-360 | 0.00 percentage of days |
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Infantile SMA Onset CS3A | Percent of Time in Hospitalization | Day 361-720 | 0.28 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 1-360 | 1.11 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 361-720 | 2.08 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous Control | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1-360 | 0.28 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 361-720 | 0.56 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1-360 | 0.28 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 361-720 | 0.00 percentage of days |
| Infantile SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 361-720 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 2 | Percent of Time in Hospitalization | Day 1-360 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 1-360 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Later SMA Onset CS12 Type 3 | Percent of Time in Hospitalization | Day 361-720 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 361-720 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 1-360 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous Control | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1-360 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 361-720 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1081-1440 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 721-1080 | 0.00 percentage of days |
| Later SMA Onset CS4 Previous ISIS 396443 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1801-2160 | 0.00 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1-360 | 0.0 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1441-1800 | 0.00 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 721-1080 | 0.0 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 361-720 | 0.0 percentage of days |
| Later SMA Onset 232SM202 | Percent of Time in Hospitalization | Day 1081-1440 | 0.69 percentage of days |
Secondary
Survival Rate
Survival rate was defined as the percentage of participants alive during the study and was estimated from the Kaplan Meier (KM) curve for time to death.
Time frame: MMDR Period: up to Day 1800
Population: All dosed population included all participants who received a dose of nusinersen or were in the sham treatment group. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Infantile SMA Onset CS3A | Survival Rate | 100 percentage of participants |
| Infantile SMA Onset CS3B Previous Control | Survival Rate | 79.16 percentage of participants |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Survival Rate | 87.69 percentage of participants |
| Infantile SMA Onset 232SM202 | Survival Rate | 97.61 percentage of participants |
| Later SMA Onset CS12 Type 2 | Survival Rate | 98.79 percentage of participants |
| Later SMA Onset CS12 Type 3 | Survival Rate | 100 percentage of participants |
| Later SMA Onset CS4 Previous Control | Survival Rate | 100 percentage of participants |
| Later SMA Onset CS4 Previous ISIS 396443 | Survival Rate | 91.66 percentage of participants |
| Later SMA Onset 232SM202 | Survival Rate | 100 percentage of participants |
Secondary
Total Number of Hospitalizations Due to Serious Adverse Events
Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization.
Time frame: Up to day 2520
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 2 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 6 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 5 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 6 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 1 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 0 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 4 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 10 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 10 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 29 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 26 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 8 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 19 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 1 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 19 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 32 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 14 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 36 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 89 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 64 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 63 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 3 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 12 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 0 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 7 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 4 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 4 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 3 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 7 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 2 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 2 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 2 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 5 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 2 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 2 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 6 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 10 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 5 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 6 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 21 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 10 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 2161-2520 | 2 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 8 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 18 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 12 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 21 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 10 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 13 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 721-1080 | 1 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1801-2160 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 361-720 | 2 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1-360 | 3 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Adverse Events | Day 1081-1440 | 3 number of hospitalizations |
Secondary
Total Number of Hospitalizations Due to Serious Respiratory Events
Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization.
Time frame: Up to day 2520
Population: The safety analysis set included all participants who were enrolled and received at least 1 dose of nusinersen or underwent sham procedure during CS11. 'Overall number of participants analyzed' signifies number of participants with data available for outcome measure analysis. 'Number analyzed' signifies number of participants with data available for analysis at specified timepoint.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 1 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 0 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 2 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 2 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 1 number of hospitalizations |
| Infantile SMA Onset CS3A | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 2 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 12 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 0 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 2 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 15 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 13 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 1 number of hospitalizations |
| Infantile SMA Onset CS3B Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 1 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 2 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 7 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 30 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 18 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 12 number of hospitalizations |
| Infantile SMA Onset CS3B Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 4 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 1 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 0 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 2 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 1 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 2 number of hospitalizations |
| Infantile SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 1 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 1 number of hospitalizations |
| Later SMA Onset CS12 Type 2 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 0 number of hospitalizations |
| Later SMA Onset CS12 Type 3 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 2 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 2 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous Control | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 3 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 2161-2520 | 0 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 1 number of hospitalizations |
| Later SMA Onset CS4 Previous ISIS 396443 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 2 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 361-720 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 721-1080 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1801-2160 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1081-1440 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1441-1800 | 0 number of hospitalizations |
| Later SMA Onset 232SM202 | Total Number of Hospitalizations Due to Serious Respiratory Events | Day 1-360 | 0 number of hospitalizations |