Contemporary cardiac issues in Duchenne muscular dystrophy

EM McNally, JR Kaltman, DW Benson, CE Canter… - Circulation, 2015 - Am Heart Assoc
EM McNally, JR Kaltman, DW Benson, CE Canter, LH Cripe, D Duan, JD Finder, WJ Groh…
Circulation, 2015Am Heart Assoc
Muscular Dystrophy, convened a Working Group meeting on July 2014, in Bethesda, MD, to
explore clinical and research questions related to cardiac disease in patients with DMD. As
a result of respiratory support and glucocorticoid use, patients with DMD are living longer,
bringing the associated cardiomyopathy to the forefront of management concern for
Duchenne patients as they age. A number of experimental agents targeted to the skeletal
muscle phenotype are in clinical trials for DMD, and the effect of these compounds on …
Muscular Dystrophy, convened a Working Group meeting on July 2014, in Bethesda, MD, to explore clinical and research questions related to cardiac disease in patients with DMD. As a result of respiratory support and glucocorticoid use, patients with DMD are living longer, bringing the associated cardiomyopathy to the forefront of management concern for Duchenne patients as they age. A number of experimental agents targeted to the skeletal muscle phenotype are in clinical trials for DMD, and the effect of these compounds on cardiac function has not been well characterized. The Working Group consisted of experts in cardiology, pulmonology, neurology, product regulation, and patient advocacy. The Working Group made clinical and research recommendations that are based on the current literature and expert opinion. Furthermore, the Working Group identified gaps in knowledge to be filled through ongoing efforts of the research and clinical communities. These recommendations to improve the diagnosis and management of cardiomyopathy in DMD included the use of CMR in clinical care and research, particularly clinical trials; the need for updated protocols to evaluate the cardiac condition in preclinical models; and the development of coordinated international biorepositories to advance treatment and research for this rare genetic disease (Table).
Am Heart Assoc