Clinical trial
Pediatric Spinal Muscular Atrophy (SMA) China Registry
Abstract
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). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.
Topics & conditions
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Keywords
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