This retrospective medical chart review aims to describe the clinical outcomes, clinical characteristics and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with the gene therapy onasemnogene abeparvovec (OA). It uses existing electronic medical record data from a single center in Saudi Arabia; no new treatment is given. Inclusion requires a genetically confirmed SMA type 1 diagnosis, OA treatment starting in January 2023 or later, and at least 3 months of follow-up after treatment.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion criteria: 1. Patients with a genetically confirmed diagnosis of SMA type 1 who were treated with OA. 2. Availability of data on at least one visit before treatment initiation and two visits post-treatment is a must for inclusion. 3. Patients who were treated with OA starting in January 2023. 4. Patients with at least 3 months of follow-up following the treatment with OA. Exclusion criteria: 1\. Any patient who does not fulfill any of the inclusion criteria listed above.