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Long-term follow-up of AVXS-101 (onasemnogene abeparvovec) in patients with spinal muscular atrophy
2019-002611-26Phase 3Unknown
- Sponsor
- AveXis
- Start
- Dec 2019
- 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 AveXis. Primary endpoint: Developmental milestone attainment. Status: unknown.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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