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Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Sponsor: Lantu Biopharma
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.
Key Details
Gender
All
Age Range
6 Months - Any
Study Type
INTERVENTIONAL
Enrollment
15
Start Date
2025-10-15
Completion Date
2030-10-15
Last Updated
2025-12-04
Healthy Volunteers
No
Conditions
Interventions
vesemnogene lantuparvovec
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Locations (1)
Tzu chi hospital
Jakarta, Indonesia, Indonesia