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
Single group
vesemnogene lantuparvovec
Biological/VaccineExploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
Participants are monitored for safety from baseline up to the end of the follow-up period.
Change from baseline in developmental gross motor milestones achieved according to WHO criteria
For patients with SMA, the percentage of participants who are able to preserve ambulatory function.
Event-free Survival until the 5th year or the last follow-up
Event-free survival was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study until the 5th year or the last follow-up.
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