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Rare Gene Mutations Inspire New Heart Drugs - The New York Times

posted onMay 25, 2017
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Article snippet: What if you carried a genetic mutation that left you nearly impervious to heart disease? What if scientists could bottle that miracle and use it to treat everyone else? In a series of studies, the most recent published on Wednesday, scientists have described two rare genetic mutations that reduce levels of triglycerides, a type of blood fat, far below normal. People carrying these genes seem invulnerable to heart disease, even if they have other risk factors. Drugs that mimic the effects of these mutations are already on the way, and many experts believe that one day they will become the next blockbuster heart treatments. Tens of millions of Americans have elevated triglyceride levels. Large genetic studies have consistently suggested a direct link to heart disease. Added to the existing arsenal of blood pressure medications, the new medications “will drive the final nail in the coffin of heart disease,” predicted Dr. John Kastelein, a professor of vascular medicine at the University of Amsterdam who was not involved in the new research. These experimental triglyceride-reducers are in early stages of development, however, and human trials have only just begun. At the moment, the optimism of researchers is rooted less in clinical trial data than in the fact that nature has produced strong evidence they should work. People like Anna Feurer may be walking proof. In 1994, Mrs. Feurer, then 40, attended a health fair held by her employer, Ralston Purina, in St. Louis.... Link to the full article to read more

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