Article

Safety and Efficacy of Vesemnogene Lantuparvovec, an AAV-Based Gene Therapy in Young Children Under 24 Months with Spinal Muscular Atrophy in Low- and Middle-Income Countries. Second Interim Report (August 2025)

2025-04-14

Abstract excerpt

<h4>Introduction</h4> Spinal muscular atrophy (SMA) is a monogenic neuromuscular disorder due to the survival motor neuron 1 mutation. Onasemnogene abeparvovec is a U.S. FDA approved single-dose gene therapy for SMA, but it is priced at USD 2.13 million per patient which severely limits its accessibility in low- and middle-income countries (LMICs). We conducted a phase 1 trial on vesemnogene lantuparvovec, an aff...

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Literature Corpus work: 42bef7a6-30e3-50bf-a3e0-b71e0e069f5c
DOI: 10.1101/2025.04.13.25325764

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