Funding outcome

Heart failure is a common and devasting condition with high morbidity and mortality. Although there have been significant advances in the therapy of heart failure in recent decades, such as the introduction of drugs that block certain pathways (beta-blockers and ACE inhibitors}, there is still a major unmet need for better therapies for many pediatric patients with heart failure. However, disappointment related to late-stage clinical failures of a number of new agents has reduced the drive for further drug development in this field. We will embark on a complete set of experimental approaches in order to enhance our understanding of the underlying disease mechanisms and targets, and highlight key issues that need to be addressed to improve the chances of success of novel therapies directed against these targets. We aim to develop potential new drugs which can be used to reduce the morbidity (suffering} and mortality (death} inflicted by heart failure. At the time of a heart transplant, our team collects various types of tissues from the diseased explanted heart. We will study how the molecules and cells are altered in these tissues and we will create new ways to reverse and/or treat these defects. We will collect various types of clinical data from these patients so that our molecular and cellular findings can be correlated with the clinical findings obtained thereby providing a unique phenotype-genotype correlation. Our findings can be directly related to heart failure in children and we hope that our discoveries will allow us to lessen the burden of heart failure.