Genetic & Genomic MedicinemedRxivPreprint
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…·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.
DOI 10.64898/2026.05.27.26354188