Cardiac pathophysiology and the future of cardiac therapies in duchenne muscular dystrophy

Tatyana A. Meyers, De Wayne Townsend

Research output: Contribution to journalReview articlepeer-review

83 Scopus citations


Duchenne muscular dystrophy (DMD) is a devastating disease featuring skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. Historically, respiratory failure has been the leading cause of mortality in DMD, but recent improvements in symptomatic respiratory management have extended the life expectancy of DMD patients. With increased longevity, the clinical relevance of heart disease in DMD is growing, as virtually all DMD patients over 18 year of age display signs of cardiomyopathy. This review will focus on the pathophysiological basis of DMD in the heart and discuss the therapeutic approaches currently in use and those in development to treat dystrophic cardiomyopathy. The first section will describe the aspects of the DMD that result in the loss of cardiac tissue and accumulation of fibrosis. The second section will discuss cardiac small molecule therapies currently used to treat heart disease in DMD, with a focus on the evidence supporting the use of each drug in dystrophic patients. The final section will outline the strengths and limitations of approaches directed at correcting the genetic defect through dystrophin gene replacement, modification, or repair. There are several new and promising therapeutic approaches that may protect the dystrophic heart, but their limitations suggest that future management of dystrophic cardiomyopathy may benefit from combining gene-targeted therapies with small molecule therapies. Understanding the mechanistic basis of dystrophic heart disease and the effects of current and emerging therapies will be critical for their success in the treatment of patients with DMD.

Original languageEnglish (US)
Article number4098
JournalInternational journal of molecular sciences
Issue number17
StatePublished - Sep 1 2019

Bibliographical note

Funding Information:
Funding: This research was funded by National Institutes of Health, grant number R01-HL114832 to D.T. and F31-HL139093 to T.A.M.

Publisher Copyright:
© 2019 by the authors.


  • Duchenne muscular dystrophy
  • Dystrophic cardiomyopathy
  • Exon skipping
  • Fibrosis
  • Gene therapy
  • Heart disease

PubMed: MeSH publication types

  • Review
  • Journal Article


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