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Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies

2 Feb 2017

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disease that primarily affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein found in myocytes, leading to a loss of muscle support and integrity. Corticosteroids are the standard supportive treatment for DMD; however, there is a high demand to expand the number of safe, effective pharmacologic options. Recently a...

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PMID: 28152217
DOI: 10.1002/phar.1909

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