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Open-Label Safety, Tolerability, and PK/PD Study of Risdiplam in SMA
2023-506739-14-00Phase 2Completed
- Sponsor
- Roche
- Start
- Dec 2023
- 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: Incidence and severity of adverse events and serious adverse events, Incidence of treatment discontinuations due to adverse events and 9 more. Started Dec 2023; status: completed.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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