Pediatric Spinal Muscular Atrophy (SMA) China Registry

Study Summary

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.

Conditions

Muscular Atrophy, Spinal

Interventions / Drugs

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment600 participants

Primary Completion—

Lead SponsorBiogen

Data Retrieved2026-04-11 02:55:21.687143+00:00

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