Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.
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Gowda V, et al. Among authors: sheehan j.
Lancet Reg Health Eur. 2023 Dec 11;37:100817. doi: 10.1016/j.lanepe.2023.100817. eCollection 2024 Feb.
Lancet Reg Health Eur. 2023.
PMID: 38169987
Free PMC article.