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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).

Ngu, L. H. et al. · 2025
bioRxiv / medRxiv · Papers · License: Open Access · 2025
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genetic and genomic medicine
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