This article includes discussion of onasemnogene abeparvovec, AVXS-101, onasemnogene, onasemnogene abeparvovec, onasemnogene abeparvovec-xioi, and Zolgensma. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.
Historical note and terminology
Onasemnogene abeparvovec is a gene therapy for delivery of a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy type 1 and is the first gene therapy to be approved for the treatment of spinal muscular atrophy in the United States in May 2019 (Hoy 2019). It was approved by the European Commission in May 2020. It will compete with intrathecal nusinersen, a splice-modulating antisense oligonucleotide, approved for the treatment of spinal muscular atrophy type 1 in 2016.
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