Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
JAMA Neurol. 2021 Jul 1;78(7):834-841. doi: 10.1001/jamaneurol.2021.1272.
JAMA Neurol. 2021.
PMID: 33999158
Free PMC article.
Clinical Trial.