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PUPFISH: Risdiplam in Infants With Spinal Muscular Atrophy
NCT05808764Phase 2Completed
- Sponsor
- Roche
- Start
- Apr 2024
- Primary completion
- May 2026
- Modality
- Small Molecules
- Countries
- 8
01
Overview
Phase 2, open-label, single-arm trial of risdiplam in spinal muscular atrophy (SMA), sponsored by Roche. Primary endpoints: Plasma Concentrations of Risdiplam, Area Under the Plasma Concentration-Time Curve of Risdiplam and 5 more. Started Apr 2024; status: completed.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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