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](https://clinicaltrials.gov/ct2/show/NCT07737756) Novartis Reference Number:COAV101A1SA01 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") 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. Condition Spinal Muscular Atrophy Overall Status Recruiting Number of Participants6 Start Date Aug 25, 2026 Completion Date Nov 25, 2026 Gender All Age(s) 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. Saudi Arabia #### Novartis Investigative Site Recruiting Dammam,15215,Saudi Arabia ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: