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

Más betegség

• 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.

• 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