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Medical Article
Introduction Duchenne muscular dystrophy (DMD) is a progressive X-linked neuromuscular disorder caused by mutations in the dystrophin gene, resulting in absence of functional dystrophin protein and progressive muscle degeneration (1). The disease affects approximately 1 in 3,500–5,000 male births and is characterized by progressive weakness, loss o

Exon-Skipping Therapy in Duchenne Muscular Dystrophy: Clinical Evidence and Functional Outcomes
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