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.
Willing and able to give written informed consent for participation in the study.
Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
None