For Healthcare Professionals

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:


Key Inclusion Criteria:

  1. 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.
  2. Genetically confirmed 5q-SMA.
  3. Age < 18 years at enrollment.


Key Exclusion Criteria:

  1. Unable or unwilling to provide informed consent.
  2. Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined inclusion/ exclusion criteria may apply.

pin location

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


Muscular Atrophy, Spinal

Age (in years)

< 18

Participants needed


Est. Completion Date

Sep 2, 2027

Treatment type

Observational [Patient Registry]


Biogen identifier


Study number


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