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Single-dose AVXS-101 in pre-symptomatic infants with spinal muscular atrophy and multiple SMN2 copies
2017-004087-35Phase 3Terminated
- Sponsor
- AveXis
- Start
- Apr 2018
- 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 endpoints: Safety and Efficacy in patients with 2 copies of SMN2. Started Apr 2018; status: terminated.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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