Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
Key Inclusion Criteria:
-Genetically confirmed 5q-SMA.
Key Exclusion Criteria:
-Other types of SMA (non 5q-SMA).
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply