Twelve-Month Outcomes of Intrathecal Vesemnogene Lantuparvovec for Spinal Muscular Atrophy in Children Younger than 24 Months in Low- and Middle- Income Countries
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 study evaluates the 12-month outcomes of vesemnogene lantuparvovec, an intrathecal gene therapy for SMA in children under 24 months in LMICs, showing safety and motor milestone improvements.
medRxiv · May 30, 2026 · PreprintGenetic & Genomic Medicine