Health Condition 1: G129- Spinal muscular atrophy, unspecified
Conditions
Interventions
Intervention1: Evrysdi®Risdiplam: Evrysdi® risdiplam powder for oral solution 0.75 mg/mL. It is prescribed orally once daily after a meal or
breastfeeding as per the routine clinical practice and Pre
Sponsors
Roche Products India Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1. Have signed the informed consent/assent (as applicable). In the case of minors, a parent or authorized adult will be required to sign the Informed Consent Form on behalf of the patient. The informed consent/assent will be obtained in accordance with local requirements 2. Patients aged =2 months at enrollment (for preterm infants this is the corrected age) 3. Confirmed diagnosis of 5q autosomal recessive SMA including genetic confirmation of homozygous deletion or compound heterozygosity that is predictive of loss of function of the survival motor neuron 1 (SMN1) gene and/or SMN2 gene 4. Clinical history, signs or symptoms attributable to SMA in patients of age 2 months and older 5. Patients who have been prescribed and received at least one dose of risdiplam for SMA.
Exclusion criteria
Exclusion criteria:
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of the study is to assess the safety and effectiveness of risdiplam for the treatment of SMA in patients of age 2 months and older in India.Timepoint: Three years | — |
Secondary
| Measure | Time frame |
|---|---|
| To describe the profile of SMA patients (2 months of age and older) in India based on demographics and familial, clinical, and genetic characteristics.Timepoint: Three years | — |
Countries
India
Contacts
Public ContactSharad Junnare
Roche Products (India) Private Limited
Outcome results
None listed