Article

Therapeutic Efficacy and Safety of onasemnogene abeparvovec Gene Therapy for Spinal Muscular Atrophy Type 1: Updated Systematic Review with Meta-Analysis

2025-11-06

Abstract excerpt

<title>Abstract</title> <p>Objectives: The objective of this study is to conduct a meta-analysis to evaluate the efficacy and safety of onasemnogene abeparvovec (OA). <h4>Background:</h4> Onasemnogene abeparvovec is a gene therapy approved from the U.S. Food and Drug Administration in May 2019. Unlike other therapies, it offers unique benefit of a single-time dose administration. The therapy has since been approv...

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Literature Corpus work: d11991e5-e63b-5d48-a0d3-74cb534b2393
DOI: 10.21203/rs.3.rs-8032586/v1

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