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Promising new drug may stop, and even cure, Huntington's disease

After an early phase clinical trial, the drug called IONIS-HTTRx, inhibited nerve-cell killing proteins

Published Wed, Dec 13, 2017 · 09:50 PM
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Washington

THE discovery of a drug that may stop, and perhaps even cure the fatal disease known as Huntington's, is being hailed as "historic" by Louise Vetter, president and CEO of the Huntington's Disease Society of America, and "phenomenal" and "fantastically promising" by Huntington's researchers, including the woman who discovered the genetic mutation that causes the disease.

"I'm ecstatic," said Professor Nancy Wexler, who in 1993 identified the mutation. "Huntington's is horrible, one of the worst diseases known to mankind, and certain death... We know it's a bad gene, making a bad protein, that makes people sick, that kills your brain cells. Anything that could impact that, we knew that there could be a cure."

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