A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

Inclusion Criteria:

• Subject's parent or legal guardian has provided signed eICF.

• Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1gene, 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 inthis 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 contactsthrough the course of the study

Exclusion Criteria:

• Patients currently enrolled in any interventional clinical trial** other than thephase IV OFELIA trial will be excluded from the study.

• Subjects who were enrolled in a clinical trial (independently of the diseaseindication and interventional treatment) but are not currently enrolled, can beincluded in this study.

During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.