Experimental drug shows potential for hard-to-treat heart disease

An experimental drug has been shown to improve symptoms, exercise capacity and heart failure status in patients with an inherited heart disease, according to a pair of newly published studies.

The studies, published in The New England Journal of Medicine and Circulation, evaluated aficamten, a next-generation cardiac myosin inhibitor, in patients with symptomatic nonobstructive hypertrophic cardiomyopathy, or nHCM. Myosin is a protein in the contractile unit of the heart muscle.

Nonobstructive HCM (nHCM) affects about 30% of people with hypertrophic cardiomyopathy, a genetic condition in which the heart muscle becomes abnormally thick and stiff. Patients often experience shortness of breath, exercise intolerance and reduced quality of life.

In The New England Journal of Medicine study, investigators gave either aficamten or a placebo to 517 trial participants and found that the drug significantly improved patient-reported health status and exercise capacity compared with placebo.

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