CARDIAC OUTCOME MEASURES IN CHILDREN WITH MUSCULAR DYSTROPHY

Baylor College Of Medicine, Houston TX

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Abstract

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. Cardiomyopathy causes significant morbidity and mortality in multiple forms of muscular dystrophy affecting children, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and subtypes of autosomal recessive limb-girdle muscular dystrophy (LGMD2). Pharmaceutical treatments for the cardiomyopathy of muscular dystrophy, including angiotensin-converting enzyme (ACE) inhibition and beta-adrenergic receptor blockade, afford significant benefit and demonstrate cardiac remodeling in clinical studies. Further studies are needed to identify and characterize more sensitive indicators of cardiac dysfunction in muscular dystrophy patients to better stratify subjects for entry into clinical protocols. Using the framework of the Cooperative International Neuromuscular Research Group (CINRG) and the Clinical and Translational Science Award (CTSA) consortium, this study will develop cardiac outcome measures that can be reliably implemented across a consortium of clinical sites devoted to the study of pharmaceutical treatments for muscular dystrophy.