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

Inclusion Criteria

• •1. Willing and able to give written informed consent for participation in the study.
• •2. Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
• •3. SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
• •4. Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
• •5. Total AAV antibody titres \< 1:20 as determined by ELISA assay.
• •6. Normal liver function (AST/ALT \< 3XULN, Bilirubin \<3.0 mg/dL).
• •7. Unable to access or failure to respond to currently available curative treatments for SMA.