Duchenne muscular dystrophy is an X-linked progressive myopathy caused by loss-of-function mutations in the dystrophin gene, primarily affecting males with onset in early childhood. Corticosteroids remain the standard of care for slowing decline, while exon-skipping therapies, gene replacement with micro-dystrophin constructs, and stop-codon read-through agents represent the leading therapeutic strategies. Multidisciplinary care has significantly extended life expectancy in recent decades.
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