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:
Exclusion criteria:
1. Any patient who does not fulfill any of the inclusion criteria listed above.
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Patients with SMA type 1 who were treated with OA.
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