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Risdiplam in presymptomatic infants with genetically diagnosed spinal muscular atrophy
2018-002087-12Phase 2Active
- Sponsor
- Roche
- Start
- Oct 2018
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of risdiplam in spinal muscular atrophy (SMA), sponsored by Roche. Primary endpoint: Proportion of infants sitting without support. Started Oct 2018; status: active.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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