Key result
Gene therapy shows promise for ambulation in Duchenne muscular dystrophy, but its effects on alleviating DMD-associated cardiomyopathy and respiratory failure remain largely unknown.
Why the study?
Cardiopulmonary failure is the most common cause of mortality in DMD patients, but little is known regarding the prospects of gene therapy on alleviating DMD-associated cardiomyopathy and respiratory failure.
Does gene therapy alleviate cardiomyopathy and respiratory failure in patients with Duchenne muscular dystrophy?
Does gene therapy alleviate cardiomyopathy and respiratory failure in patients with Duchenne muscular dystrophy?
This review highlights the need to evaluate the effects of emerging gene therapies on cardiopulmonary outcomes, the leading cause of mortality in Duchenne muscular dystrophy.
Answers are grounded in this paper and cite their sources.
Cardiopulmonary effects of DMD gene therapy remain unknown; leaves open whether therapies address leading mortality drivers.
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 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.
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McKim et al. (2020) conducted a review in Duchenne muscular dystrophy. Gene therapy was evaluated. Gene therapy shows promise for ambulation in Duchenne muscular dystrophy, but its effects on alleviating DMD-associated cardiomyopathy and respiratory failure remain largely unknown.
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