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New heart disease mechanism revealed: Next-generation targeted therapy shows benefit across mutation types

A study led by the Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), working in collaboration with an international research team, has identified a new molecular mechanism involved in hypertrophic cardiomyopathy, the most common inherited cardiovascular disease.

The research, published in Nature Cardiovascular Research, also demonstrates that mavacamten—the first targeted therapy available for this condition—is effective across different types of genetic mutations.

Hypertrophic cardiomyopathy is the most common inherited heart disease and the leading cause of sudden cardiac death in young people and athletes.

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