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Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy.
Stevens D, Claborn MK, Gildon BL, Kessler TL, Walker C. Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy. Ann Pharmacother. 2020 10; 54(10):1001-1009.
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PubMed
subject areas
Child, Preschool
Drug Approval
Female
Genetic Therapy
Humans
Mutation
Recombinant Fusion Proteins
Spinal Muscular Atrophies of Childhood
Survival of Motor Neuron 1 Protein
United States
United States Food and Drug Administration
Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy.