Article

Real world safety and exploratory efficacy of gene therapy for patients with 5q-Spinal Muscular Atrophy in a Brazilian cohort

2023-02-28

Abstract excerpt

<title>Abstract</title> <p>Spinal muscular atrophy (SMA) is a motor neuron disease associated with progressive muscle weakness, ventilatory failure, and reduced survival. Onasemnogene abeparvoves is the first gene replacement therapy (GT) approved to treat this condition. An observational retrospective study was conducted to assess adverse events and efficacy of GT in SMA patients. Forty-one patients with SMA (24...

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Literature Corpus work: 4555620f-f496-5dc7-86c1-4551e64bd265
DOI: 10.21203/rs.3.rs-2527927/v1

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