The effect of emerging therapies on cardiopulmonary disease in Duchenne
muscular dystrophy
- Doug McKim,
- Timothy Cripe,
- Linda Cripe
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.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
07 Aug 2020Editorial Decision: Revise Minor
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