Clinical Research Directory

Inclusion Criteria: * Subject's parent or legal guardian has provided signed eICF. * Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene. * Subject treated\* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study. Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it. \*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements. * Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study Exclusion Criteria: * Patients currently enrolled in any interventional clinical trial\*\* other than the phase IV OFELIA trial will be excluded from the study. * Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study. During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.