Study design & results
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Trial intelligence / Profile preview
Open-label risdiplam study in previously treated patients with spinal muscular atrophy
NL-OMON49730Phase 2Recruiting
- Sponsor
- Roche
- Start
- Jun 2019
- 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 endpoints: Incidence and severity of adverse events, Treatment discontinuations due to adverse events and 13 more. Started Jun 2019; status: recruiting.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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