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Phase 3 single-arm IV AVXS-101 gene therapy trial in infants with SMA type 1
NL-OMON48542Phase 3Withdrawn
- 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 type 1, sponsored by AveXis. Primary endpoint: Sitting without support by 18 months. Planned start May 2018; status: withdrawn.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy type 1
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