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A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
Sponsor: Novartis Pharmaceuticals
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.
Official title: Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
Key Details
Gender
All
Age Range
14 Days - 2 Years
Study Type
OBSERVATIONAL
Enrollment
6
Start Date
2026-08-20
Completion Date
2026-11-20
Last Updated
2026-07-30
Healthy Volunteers
No