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Trial intelligence / Profile preview
Presymptomatic AVXS-101 in infants with SMA and multiple SMN2 copies
NL-OMON46178Phase 3Withdrawn
- Sponsor
- NA
- Start
- Feb 2019
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 3, open-label trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA) (2 arms), sponsored by NA. Planned start Feb 2019; status: withdrawn.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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