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The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy
Author(s) -
McKim Douglas A.,
Cripe Timothy P.,
Cripe Linda H.
Publication year - 2021
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.25079
Subject(s) - medicine , duchenne muscular dystrophy , muscular dystrophy , clinical trial , cardiomyopathy , disease , respiratory failure , neuromuscular disease , genetic enhancement , cardiology , intensive care medicine , heart failure , pediatrics , gene , genetics , biology
Gene therapy is an attractive approach being intensively studied to prevent muscle deterioration in patients with Duchenne muscular dystrophy. While clinical trials are only in early stages, initial reports are promising for its effects on ambulation. Cardiopulmonary failure, however, is the most common cause of mortality in Duchenne muscular dystrophy (DMD) patients, and little is known regarding the prospects for gene therapy on alleviating DMD‐associated cardiomyopathy and respiratory failure. Here we review current knowledge regarding effects of gene therapy on DMD cardiomyopathy and discuss respiratory endpoints that should be considered as outcome measures in future clinical trials.