Evaluating the Safety and Effectiveness of Vesemnogene Lantuparvovec Gene Therapy for Spinal Muscular Atrophy in Children 6 Months and Older in Low- and Middle-Income Countries

What this Trial Is About

Researchers are evaluating the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for Spinal Muscular Atrophy SMA, especially in low- and middle-income countries. The study aims to measure how well this gene therapy helps children with SMA develop gross motor skills and survive, while also tracking any side effects or adverse events that might occur. This observational study involves administering the selected dose of Vesemnogene lantuparvovec to children aged 6 months and older who have a genetic diagnosis of SMA. After treatment, participants will be closely monitored to assess their response and any possible toxicity. The study does not require participants to stay enrolled and allows them to leave at any time by simply not continuing visits. During the study, researchers will collect data on adverse events and serious adverse events from baseline until the child reaches 5 years old. They will also track changes in motor skill development based on WHO criteria and monitor survival rates up to five years or the last follow-up. Participants will undergo regular assessments, including laboratory tests, to evaluate liver function and antibody levels, ensuring careful safety monitoring throughout the study period.

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).