Journal
JACC-BASIC TO TRANSLATIONAL SCIENCE
Volume 3, Issue 3, Pages 391-402Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.jacbts.2018.03.007
Keywords
cardiomyopathy; L-type calcium channels; mitochondria
Categories
Funding
- Raine Priming (RPG50)
- Muscular Dystrophy Association USA [272200]
- National Health and Medical Research Foundation of Australia [1062740, 1043758]
- National Health and Medical Research Council of Australia [1062740] Funding Source: NHMRC
Ask authors/readers for more resources
Current clinical trials demonstrate Duchenne muscular dystrophy (DMD) patients receiving phosphorodiamidate morpholino oligomer (PMO) therapy exhibit improved ambulation and stable pulmonary function; however, cardiac abnormalities remain. Utilizing the same PMO chemistry as current clinical trials, we have identified a non-toxic PMO treatment regimen that restores metabolic activity and prevents DMD cardiomyopathy. We propose that a treatment regimen of this nature may have the potential to significantly improve morbidity and mortality from DMD by improving ambulation, stabilizing pulmonary function, and preventing the development of cardiomyopathy. (C) 2018 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available