Last Updated: 09/26/2026
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Found 4 publications
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
Journal: Journal of neuromuscular diseases
Published: August 06, 2019
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.
Journal: Pediatric neurology
Published: March 22, 2019
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: November 02, 2017
Showing 1-4 of 4
Last Updated: 09/26/2026