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Phase 3 single-arm intravenous onasemnogene abeparvovec in SMA type 1
2020-000095-38Phase 3
- Sponsor
- AveXis
- Start
- Mar 2020
- 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 type 1, sponsored by AveXis. Primary endpoint: Functional independent sitting for at least 30 seconds at 18 months of age.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy type 1
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