Neuromuscular cardiomyopathies

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

doi:10.1007/978-3-319-44577-9_12

Neuromuscular cardiomyopathies

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

Research output: Chapter in Book/Report/Conference proceeding › Chapter

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 language	English (US)
Title of host publication	Congestive Heart Failure and Cardiac Transplantation
Subtitle of host publication	Clinical, Pathology, Imaging and Molecular Profiles
Publisher	Springer International Publishing
Pages	175-196
Number of pages	22
ISBN (Electronic)	9783319445779
ISBN (Print)	9783319445755
DOIs	https://doi.org/10.1007/978-3-319-44577-9_12
State	Published - Jun 1 2017

Keywords

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

ASJC Scopus subject areas

Medicine(all)

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10.1007/978-3-319-44577-9_12

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Neuromuscular cardiomyopathies

Abstract

Keywords

ASJC Scopus subject areas

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