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[Comment] Treatment of Duchenne muscular dystrophy: first small steps

Duchenne muscular dystrophy (DMD) is a progressive and life-limiting X-linked recessive disorder caused by mutations in the DMD gene that result in reduced or absent dystrophin production. Dystrophin is part of the dystrophin–glycoprotein complex, which acts as a scaffold between the actin cytoskeleton and the extracellular matrix and, as such, maintains muscle fibre integrity. Absence of dystrophin causes progressive and irreversible tissue damage, resulting in muscle fibrosis and fatty replacement.

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