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IV OAV101 gene replacement therapy in pediatric spinal muscular atrophy
2020-005995-37Phase 3Completed
- Sponsor
- Novartis
- Start
- Jun 2021
- 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 endpoint: Treatment-emergent adverse events and serious adverse events. Started Jun 2021; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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Study design, results and competitive context are available in the Gosset platform where reported.
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