"This is the first gene-targeted medicine in cardiovascular disease, " says Cleveland Clinic's Topol, who is helping design the trial.

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Because the pain medicine is so specifically targeted, it can be delivered in doses of a thousandth to a ten-thousandth of those currently employed.

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The new medicine, eteplirsen, is targeted at the 13% of those boys whose muscular dystrophy is caused by a certain type of mutation.

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The research should also help speed the development of other gene-targeted drugs for cancer, says Haber, senior author on the New England Journal of Medicine paper and director of the Massachusetts General Hospital Cancer Center.

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