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Risdiplam after gene therapy plateau or decline in pediatric spinal muscular atrophy
2023-505161-81-00Phase 4Authorised
- Sponsor
- Roche
- Start
- Dec 2024
- Modality
- Small Molecules
- Countries
- 6
01
Overview
Phase 4, open-label, single-arm trial of risdiplam in spinal muscular atrophy (SMA), sponsored by Roche. Primary endpoint: Change from baseline in BSID-III gross motor raw score at Week 72. Planned start Dec 2024; status: authorised.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
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