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RECRUITING
Updated: Sep 22, 2026
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia (SMA-REAL)
Brief Summary
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.
Key Information
Participation Criteria
Study Design
Locations & Contacts
Inclusion Criteria:
- 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.
Exclusion Criteria:
1. Any patient who does not fulfill any of the listed above.