Dilated cardiomyopathy (DCM) is a prevalent cause of heart failure in children and is the most common indication for heart transplantation in the pediatric population. Currently, no effective strategies exist to predict outcomes or adequately treat these individuals. These finding highlight the clinically unmet need to identify new therapies for individuals afflicted with this devastating disease, a core mission of the CDI.
The objective of this large-scale interdisciplinary research initiative is to test the hypothesis that application of aprecision medicine paradigm based on genetic testing can resolve distinct subtypes of pediatric DCM with unique clinical phenotypes, patient outcomes, and molecular mechanisms of disease. Furthermore, we aim to develop mutation-specific therapeutics using novel experimental platforms.
· Define Clinical Subtypes of Pediatric Dilated Cardiomyopathy.
· Identify Mechanisms that are Responsible for Disease Initiation.
· Develop Precision Therapeutics for Pediatric Dilated Cardiomyopathy.
This initiative will establish the applicability of precision medicine to pediatric DCM and result in paradigm shifts that will reshape our approach towards treating heart failure in children, including the development of new diagnostics and therapeutics.