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SPR1NT presymptomatic intravenous AVXS-101 study in spinal muscular atrophy with multiple SMN2 copies
MOH_2019-07-15_003587Phase 3Not yet recruiting
- Sponsor
- AveXis
- Start
- May 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. Planned start May 2018; status: not yet recruiting.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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