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

NCT07737756

Not Yet RecruitingN/A

Sponsor: Novartis Pharmaceuticals

Conditions: Spinal Muscular Atrophy

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.

Eligibility overview

Sex: ALL

Age: 14 Days to 2 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

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.