Neuromuscular cardiomyopathies

Forum Kamdar, Pradeep P.A. Mammen, Daniel J. Garry

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

More than 30 types of muscular dystrophies are included in the broader group of neuromuscular cardiomyopathies. Skeletal muscle weakness is a defining characteristic of muscular dystrophy, but as treatments improve and patients live longer, cardiomyopathy has emerged as the leading cause of death for patients with Duchenne or Becker muscular dystrophy. This chapter focuses on Duchenne and Becker muscular dystrophies, from their genetic origins, symptoms and disease progression to cardiac involvement and treatments, as well as research findings. Promising emerging therapies include exon skipping, dystrophin mini-gene replacement, sealants, and gene-editing strategies.

Original languageEnglish (US)
Title of host publicationCongestive Heart Failure and Cardiac Transplantation
Subtitle of host publicationClinical, Pathology, Imaging and Molecular Profiles
PublisherSpringer International Publishing
Pages175-196
Number of pages22
ISBN (Electronic)9783319445779
ISBN (Print)9783319445755
DOIs
Publication statusPublished - Jun 1 2017

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Keywords

  • Becker muscular dystrophy
  • Duchenne muscular dystrophy
  • Dystrophin gene
  • Dystrophinopathies
  • Exon skipping
  • Gene editing
  • Neuromuscular cardiomyopathies

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Kamdar, F., Mammen, P. P. A., & Garry, D. J. (2017). Neuromuscular cardiomyopathies. In Congestive Heart Failure and Cardiac Transplantation: Clinical, Pathology, Imaging and Molecular Profiles (pp. 175-196). Springer International Publishing. https://doi.org/10.1007/978-3-319-44577-9_12