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OAV101 intrathecal after discontinuation of nusinersen or risdiplam in pediatric SMA
2021-006709-31Phase 3Active
- Sponsor
- Novartis
- Start
- Jul 2022
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoints: Adverse events over 52 weeks, Related adverse events over 52 weeks and 2 more. Started Jul 2022; status: active.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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