A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Participant's age at life-altering clinical milestones- failure to self-feed during 30 months of this study was analyzed using the Kaplan-Meier method. Age was summarized in years.

Time frame: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, 'Number of Participants Analyzed' signifies participants included in the Kaplan-Meier analysis. No participant in Ambulatory participants aged \<6 years and \>=6 years had an event of failure to self-feed.

Participant's age at life-altering clinical milestones- failure to stand was calculated based on the birthdate and the date of failure to stand as reported by caregiver during 30 months of this study. Participants who were not reported being failure to stand by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

Time frame: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, 'Number of Participants Analyzed' signifies participants included in the Kaplan-Meier analysis. No participant in Ambulatory participants aged \<6 years had an event of failure to stand.

Participant's age at life-altering clinical milestones- failure to walk was calculated based on the birthdate and the date of failure to walk as reported by caregiver during 30 months of this study. Participants who were not reported being failure to walk by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

Time frame: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, 'Number of Participants Analyzed' signifies participants included in the Kaplan-Meier analysis. No participant in Ambulatory participants aged \< 6 years had an event of failure to walk.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 (normal- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (example \[e.g.\] standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 (normal- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 (normal- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 (normal- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 (normal- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Forced expiratory volume in one second (FEV1) is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Forced vital capacity (FVC) is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. Number Analyzed signifies participants evaluable for the specified rows.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows.

WISC-IV is an individually administered intelligence test for children between the ages of 6 and 16. The WISC-IV Composites are: Verbal Comprehension, Perceptual Reasoning, Working Memory, and Processing Speed. Scores from the Composites constitute the WISC-IV Full Scale IQ score which ranges from 40 (Exceptionally Low) to 160 (Exceptionally Superior), higher scores indicated more intelligence. The WISC was only performed in ambulatory participants \>= 6 to \<=16 years old as pre-specified in the protocol.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of severity: 1=no problems, 2=some problems and 3=extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00. Higher scores indicating a better quality of life. EQ-5D-3L was only performed in participants \>= 16 years old.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \>=16 years. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state), where higher scores indicated better quality of life. EQ-VAS assessment was only performed in the participants \>= 16 years old.

Time frame: Baseline (Day 1) and Months 12, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \>=16 years. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<=16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems. Participants indicated their health state by choosing the appropriate level from each dimension. The 5 digit health states thus obtained for each dimension were then converted into a single median index value. A health state index score was calculated from individual health profiles using a USA scoring algorithm. Health state index scores generally ranged from -0.109 to 1, where, -0.109= the worst health status, 1= full health. Higher the score the better the better quality of life.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \<16 years and contributed data to table, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \<16 years and contributed data to table, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to their child's use of healthcare professionals, emergency room visits, and hospitalizations in past 3 months. Change from baseline in mean number of visits to primary care physician, emergency room and office visits is presented in this outcome measure.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Number of Participants Analyzed' signifies all participants in the Full Analysis Set. All participants reported under Number of Participants Analyzed contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to number of nights in hospital due to disease/dedication for disease in past 3 months. Change from baseline in mean number of nights in hospital due to disease or medication for disease is presented in this outcome measure.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Caregivers were asked to estimate out-of-pocket costs related to healthcare resource utilization. The number of out of pocket money was defined as the total spent of the past three months in managing child's Duchenne muscular dystrophy.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

Time frame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies all participants in the Full Analysis Set with age \<=10 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Time frame: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Time frame: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Time frame: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Time frame: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies participants evaluable for this outcome measure.

The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

Time frame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies all participants in the Full Analysis Set with age between 11-18 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

Time frame: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, Number of Participants Analyzed signifies all participants in the Full Analysis Set with age between 11-18 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.