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Article

New and emerging pharmacotherapy for duchenne muscular dystrophy: a focus on synthetic therapeutics.

Expert opinion on pharmacotherapy - 1 May 2020

Grages Sharon M, Bell Michael, Berlau Daniel J

Abstract excerpt

INTRODUCTION: Duchenne muscular dystrophy (DMD) is the result of X-chromosome-linked mutations to the dystrophin protein gene that prevent the normal development and repair of muscles leading to muscle deterioration. The condition affects nearly 1 in 3,500 males worldwide. Current therapeutics have not been sufficient in providing a cure or resulting in a significant extension in life expectancy, but many...

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Identifiers

PMID: 32133879
DOI: 10.1080/14656566.2020.1732350

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