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

Summary

The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival. * To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.

Timeline

Start

2025-10-15

Primary completion

2030-10-15

Completion

2030-10-15

Drugs

Indications