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Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy
ChiCTR2300072288Phase 4Completed
- Sponsor
- Peking University First Hospital
- Start
- Sep 2023
- Modality
- Small Molecules
- Countries
- 1
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Overview
Phase 4, open-label trial of risdiplam in spinal muscular atrophy (SMA) (2 arms), sponsored by Peking University First Hospital. Primary endpoint: Change from baseline in the total score of Motor Function Scales. Started Sep 2023; status: completed.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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