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Open-label risdiplam study in presymptomatic infants with genetically diagnosed spinal muscular atrophy
2023-506009-20-00Phase 2Authorised
- Sponsor
- Roche
- Start
- Mar 2019
- Modality
- Small Molecules
- Countries
- 7
01
Overview
Phase 2, open-label, single-arm trial of risdiplam in spinal muscular atrophy (SMA), sponsored by Roche. Planned start Mar 2019; status: authorised.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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