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

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Conditions studied: Spinal Muscular Atrophy

In brief

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 facts

Study ID

NCT07737756

Run by

Novartis Pharmaceuticals

People needed

6

Starts

2026-08-20

Expected to finish

2026-11-20

Last updated by the study team

2026-07-30

Who can join

Age: 0 and older, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Any patient who does not fulfill any of the inclusion criteria listed above.

Full record on ClinicalTrials.gov

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