A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

The requested reference [80ab663485a5dcb2c5b93d334a108fbf6752c8c5] no longer exists in our database. It has been replaced by the current reference: [cab09fe4f737172193600c60caad87a49a188e01]

Authors
Category Primary study
Registry of TrialsClinicalTrials.gov
Year 2026
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan. This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.
Epistemonikos ID: 05a010565f77ba1c31b950f0387bfd9c373311b0
First added on: Aug 17, 2026