Last Update: Sep 22, 2026
Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
ClinicalTrials.gov Identifier:
NCT07737756
Novartis Reference Number:COAV101A1SA01
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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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.

Spinal Muscular Atrophy
Recruiting
6
Aug 25, 2026
Nov 25, 2026
All
14 Years - 2 Years (Child)

Eligibility Criteria

Inclusion criteria:

1. Patients with a genetically confirmed diagnosis of SMA type 1 who were treated with OA.
2. Availability of data on at least one visit before treatment initiation and two visits post-treatment is a must for inclusion.
3. Patients who were treated with OA starting in January 2023.
4. 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 inclusion criteria listed above.

Novartis Investigative Site

Recruiting

Dammam,15215,Saudi Arabia

Worldwide Contacts

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Novartis Pharmaceuticals

+41613241111