Status: Recruiting
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
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.
Participants needed: 15
Trial details
Phase: Phase 3Age: 6+Biological sex: AllType: InterventionalSponsor: Lantu BiopharmaUpdated: Dec 4, 2025Locations: 1
Eligibility criteria
Willing and able to give written informed consent for participation in the study... [+6]