Genetic & Genomic MedicinemedRxivPreprint
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).
Lock Hock Ngu, Qingling Mo, Shiqi Li…·Austin T. Gao
This paper reports on a phase 1 trial of vesemnogene lantuparvovec, an affordable gene therapy for SMA, showing safety and improved motor milestones in young children.
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