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The effect of emerging therapies on cardiopulmonary disease in Duchenne muscular dystrophy
  • Doug McKim,
  • Timothy Cripe,
  • Linda Cripe
Doug McKim
Ottawa Hospital Research Institute
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Timothy Cripe
Nationwide Children's Hospital
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Linda Cripe
Nationwide Children’s Hospital
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Abstract

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.

Peer review status:ACCEPTED

31 Jul 2020Submitted to Pediatric Pulmonology
04 Aug 2020Submission Checks Completed
04 Aug 2020Assigned to Editor
07 Aug 2020Reviewer(s) Assigned
07 Aug 2020Review(s) Completed, Editorial Evaluation Pending
27 Aug 20201st Revision Received
28 Aug 2020Submission Checks Completed
28 Aug 2020Assigned to Editor
28 Aug 2020Reviewer(s) Assigned
28 Aug 2020Review(s) Completed, Editorial Evaluation Pending
28 Aug 2020Editorial Decision: Accept