Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy characterized by rapid progression of muscle degeneration that eventually leads to death. However, due to advances in palliative respiratory care, heart failure is now emerging as a leading cause of death, and there are currently no effective therapies for this fatal clinical consequence of DMD. But now, DeWayne Townsend and colleagues, at the University of Minnesota, Minneapolis, have found that chronic infusion of membrane-sealing poloxamer blocks advanced heart disease in the golden retriever muscular dystrophy model of DMD. Further analysis provided a mechanistic basis for the beneficial effect of poloxamer: it restored the compliance of heart muscles from the dystrophic dogs to normal. The authors therefore suggest that membrane-sealant therapy could provide a new approach to treating DMD heart disease.
TITLE: Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs
AUTHOR CONTACT:
DeWayne Townsend
University of Minnesota, Minneapolis, Minnesota, USA.
Phone: 612.625.6873; Fax: 612.625.5149; E-mail: town0045@umn.edu .
View this article at: http://www.jci.org/articles/view/41329?key=638f71f392c753a858b7
Journal of Clinical Investigation