Cardiomyopathy of Duchenne muscular dystrophy: Pathogenesis and prospect of membrane sealants as a new therapeutic approach

Research output: Contribution to journalReview articlepeer-review

23 Scopus citations

Abstract

Duchenne muscular dystrophy (DMD) is a devastating progressive disease of striated muscle deterioration. This fatal X-linked disorder results from the loss of the protein dystrophin, which in turn causes striated muscle membrane instability. Cardiac dysfunction is a growing problem in patients with DMD, but relatively little is known about the pathophysiology of the dystrophic heart. At present, there is no effective treatment for DMD and the current clinical approaches are primarily supportive in nature. This review will discuss the pathogenesis of DMD in the heart and discuss how these pathogenic processes have led to a new class of agents directed specifically at restoring membrane integrity to dystrophic myocardium. The tri-block poloxamers, specifically poloxamer 188 (P188), are able to stabilize the membranes of dystrophic myocardium in animal models and may offer a new therapeutic approach for cardiac disease in DMD.

Original languageEnglish (US)
Pages (from-to)99-109
Number of pages11
JournalExpert review of cardiovascular therapy
Volume5
Issue number1
DOIs
StatePublished - Jan 2007

Bibliographical note

Funding Information:
This work was supported by grants from the National Institute on Aging (JMM), NIH NRSA (DT), and the Muscular Dystrophy Association (SY).

Copyright:
Copyright 2008 Elsevier B.V., All rights reserved.

Keywords

  • Cardiomyopathy
  • Duchenne muscular dystrophy
  • Membrane sealant
  • Poloxamer 188

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