A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia

The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA) at a single clinical center in Saudi Arabia. The study will use secondary data collected from the electronic medical records of SMA type 1 patients.

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.