Spotlight on Duchenne muscular dystrophy

Duchenne muscular dystrophy is a common and devastating disease affecting males. Dr. Gyula Acsadi of the University of Connecticut outlines the clinical presentation and advances in the molecular pathogenesis of Duchenne muscular dystrophy. Early recognition of cardiac and respiratory pathophysiology and proactive management, along with corticosteroid treatment, will have a significant impact on improving the outcome of Duchenne muscular dystrophy. Various genetic therapy strategies including “exon skipping” are entering into clinical trials, thus, improving the prospect for therapy for muscular dystrophies.

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