Hypertrophic cardiomyopathy (HCM) is a
genetic disorder caused by pathogenic variants in sarcomeric genes, leading to
left ventricular hypertrophy and complex phenotypic heterogeneity. While HCM is the most common inherited
cardiomyopathy, pharmacological treatment options have previously been limited and were predominantly directed towards symptom control owing to
left ventricular outflow obstruction. These
therapies, including beta
blockers, calcium channel blockers, and
disopyramide, have not been shown to affect the natural history of the disease, which is of particular concern for younger patients who have an increased lifetime risk of experiencing arrhythmias,
heart failure, and
sudden cardiac death. Increased knowledge of the genetic mechanisms underlying this disease in recent years has led to the development of targeted, potentially disease-modifying
therapies for both obstructive and nonobstructive phenotypes that may help to prevent or ameliorate
left ventricular hypertrophy. In this review article, we will define the etiology and clinical phenotypes of HCM, summarize the conventional
therapies for obstructive HCM, discuss the emerging targeted
therapies as well as novel invasive approaches for obstructive HCM, describe the therapeutic advances for nonobstructive HCM, and outline the future directions for the treatment of HCM.