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An experimental drug to treat an inherited, progressive heart disease clearly improved the symptoms of patients, its maker, MyoKardia, said Monday.

The full data have not yet been published or reviewed by outside experts. But cardiologists called the top-line results alone a big step forward for patients with the disorder, called hypertrophic cardiomyopathy, or HCM. In the disorder, the wall of the left ventricle, the chamber of the heart that pumps blood throughout the body, thickens and the heart’s pumping action becomes less effective.


MyoKardia, based in Brisbane, Calif., said that the results were statistically significant not only on the study’s main goal, but also on every other measure the company had set out to study. Its medicine, mavacamten, was tested in 251 patients with HCM who were assigned to receive either the drug or placebo.

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