A study to observe if Risdiplam treatment is safe and effective in Spinal Muscular Atrophy Patients in India.

Not Applicable

Recruiting

Brief Summary: | |

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|This is a non-interventional, single-arm, observational study with primary prospective data collection (NIS PDC) and retrospective collection of prior medical/treatment history data from medical records. Study is designed to evaluate the safety and effectiveness of risdiplam for the treatment of SMA patients in the routine clinical setting. All treatment choices in this study will be at the discretion of the treating physician according to local standard of care or best practice and are independent of participation in this study. No additional laboratory or other diagnostic tests will be performed on patients due to their participation in this study. No study-specific site visits are mandated by the study protocol.

Recruitment & Eligibility

Inclusion Criteria

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 â‰¥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.

Exclusion Criteria

Not provided

Primary Outcome Measures

Name	Time	Method
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.	Three years

Secondary Outcome Measures

Name	Time	Method
To describe the profile of SMA patients (2 months of age and older) in India based on demographics and familial, clinical, and genetic characteristics.	Three years

Locations (7): Royal Institute of Child Neuro Sciences
🇮🇳
Ahmadabad, GUJARAT, India
Aster MIMS Hospital
🇮🇳
Kozhikode, KERALA, India
Jaslok Hospital and Research Centre
🇮🇳
(Suburban), MAHARASHTRA, India
NairÂ Hospital
🇮🇳
Mumbai, MAHARASHTRA, India
P D Hinduja Hospital and Medical Research Centre
🇮🇳
(Suburban), MAHARASHTRA, India
Peerless Hospital
🇮🇳
Kolkata, WEST BENGAL, India
Sir Gangaram Hospital
🇮🇳
Delhi, DELHI, India
Royal Institute of Child Neuro Sciences
🇮🇳Ahmadabad, GUJARAT, India
Dr Siddharth Shah
Principal investigator
9909960555
sidh909@hotmail.com