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Article

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.

Expert opinion on biological therapy - 1 Sept 2022

McMillan Hugh J, Proud Crystal M, Farrar Michelle A, Alexander Ian E, Muntoni Francesco, Servais Laurent

Abstract excerpt

INTRODUCTION: Gene therapy for spinal muscular atrophy (SMA) represents a significant milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that results in motor neuron loss because of mutations of the survival motor neuron 1 gene, which directs survival motor neuron (SMN) protein production. Onasemnogene abeparvovec, a one-time gene replacement therapy, delivers a functional...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Atrophy, Spinal

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Share to Spinal Muscular Atrophies of Childhood

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Identifiers

PMID: 35437095
DOI: 10.1080/14712598.2022.2066471

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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