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, Tsun-Haw Toh, Jia Ni Lee, Kok Chong Lim, Edi Setiawan Tehuteru, Rahmi Lestari, Chinnuwat Sanguansermsri, Hany Abueita, Samson Gwer, Li Li, Zhuowei Wang, Salman Kirmani, Julia X. Chen, , 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.
medRxiv · Apr 13, 2025 · PreprintGenetic & Genomic Medicine