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.
Patients with a genetically confirmed diagnosis of SMA type 1 who were treated with OA.
Availability of data on at least one visit before treatment initiation and two visits post-treatment is a must for inclusion.
Patients who were treated with OA starting in January 2023.
Patients with at least 3 months of follow-up following the treatment with OA.
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