The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S. Pragmatic Multicenter Study (STREAM).
The participant has a genetically confirmed diagnosis of SMA (biallelic SMN1 deletion or mutation).
He or she is ≥ 2 years of age on the day of the intrathecal injection.
The treating Investigator intends to administer within the current episode of care, a single dose of ITVISMA® (1.2 × 10¹⁴ vg) in routine U.S. practice after written informed consent obtained.
independently ambulatory child/adolescent 2 - <18 years; or
Contraindication for lumbar puncture as determined by the investigator (e.g., spinal anatomy that precludes safe lumbar puncture)
An uncontrolled acute illness, active infection, febrile illness or acute medical condition, within 30 days prior to dosing
Inability to tolerate corticosteroids administered by mouth or gastrostomy tube
Current participation in another interventional clinical trial that would interfere with the objectives or endpoints of STREAM. Note: participation in observational cohort studies or non-interventional studies in which the participant does not receive treatment or undergo procedures which may compromise this study data integrity may be allowed following Sponsor approval