Browsing by Author "4e1e31b7-fd9c-43a8-a5e7-194f4dd996fa"
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McDonald, Craig M.; Campbell, Craig; Erazo Torricelli, Ricardo; Finkel, Richard S. (Elsevier, 2017)Background Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of disease; therefore, mutation-specific therapies ...