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

Novartis Pharmaceuticals · 2026
ClinicalTrials.gov · Datasets · License: Public Domain · 2026
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gene-therapyrare-diseasesspinal-muscular-atrophy
spinal muscular atrophy, neuromuscular disorders, pediatric neurology, gene therapy, onasemnogene abeparvovec, rare diseases, observational
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