Novel therapies in inherited cardiomyopathies

Abstract Despite significant advances in the field of genetic cardiomyopathies, the treatment of inherited cardiac disease has long focused on symptom management and therapies geared towards the consequences of heart failure. More recently, small molecule therapy has emerged as a targeted approach that seeks to alter the progression of genetic cardiac disease at the molecular level. Small molecule myosin inhibitors have been developed to decrease the hypercontractility observed in conditions like hypertrophic cardiomyopathy and myosin activators seek to reverse the reduced left ventricular function seen in dilated cardiomyopathy. Further targeting specific genetic etiologies of cardiomyopathy, P38α MAP kinase inhibition is a therapy developed to counter abnormally upregulated pathways in lamin A/C cardiomyopathy. While trials are still ongoing, these small molecule therapies could alter the treatment landscape of genetic cardiomyopathies for years to come.