Pediatric Spinal Muscular Atrophy (SMA) China Registry
About the study
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA).
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Key Inclusion Criteria:
- Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
- Genetically confirmed 5q-SMA.
- Age < 18 years at enrollment.
EXCLUSION CRITERIA
Key Exclusion Criteria:
- Unable or unwilling to provide informed consent.
- Other types of SMA (non 5q-SMA).
NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Muscular Atrophy, Spinal
Age (in years)
< 18
Participants needed
600
Est. Completion Date
Sep 2, 2027
Treatment type
Observational [Patient Registry]
Sponsor
Biogen
ClinicalTrials.gov identifier
NCT05042921
Study number
CN-SMG-11774
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