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Article

Cardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics, and new clinical challenges.

The Lancet. Respiratory medicine - 1 Apr 2022

Birnkrant David J, Bello Luca, Butterfield Russell J, Carter John C, Cripe Linda H, Cripe Timothy P, McKim Douglas A, Nandi Deipanjan, Pegoraro Elena

Abstract excerpt

The life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung function and progressive cardiomyopathy; when patients are treated with assisted ventilation, cardiac function becomes the main determinant of survival. Therapy for DMD is changing rapidly, with the emergence of new genetic and molecular therapeutic options, the proliferation of which has fostered the perception that DMD is...

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PMID: 35364035
DOI: 10.1016/S2213-2600(21)00581-6

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