New approaches in the therapy of cardiomyopathy in muscular dystrophy

Elizabeth M. McNally*

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingChapter

87 Scopus citations


Cardiomyopathy is a frequent occurrence in muscular dystrophy, and heart disease in muscular dystrophy can contribute to both morbidity and mortality. A number of novel therapies are being developed for muscular dystrophy, and the efficacy of these therapies for heart disease is unknown. The most common X-linked recessive disease is Duchenne muscular dystrophy (DMD), which arises from defects in the dystrophin gene. Therapy specifically aimed at DMD is reviewed in the context of its projected effect on cardiomyopathy associated with DMD. Additionally, novel therapies are being pursued to treat specifically the cardiomyopathy of DMD. There is substantial genetic heterogeneity underlying the muscular dystrophies, and not all muscular dystrophy patients develop cardiomyopathy. A subset of muscular dystrophies may place patients at significantly greater risk of developing cardiomyopathy and cardiac rhythm disturbances. These disorders are discussed, highlighting recent studies and recommendations for therapy.

Original languageEnglish (US)
Title of host publicationAnnual Review of Medicine
Editors Caskey, Austin, Hoxie
Number of pages14
StatePublished - May 11 2007

Publication series

NameAnnual Review of Medicine
ISSN (Print)0066-4219


  • Dystrophin
  • Dystrophin glycoprotein complex
  • Lamin

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)


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