Daring to hope

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Science  24 Aug 2018:
Vol. 361, Issue 6404, pp. 742-746
DOI: 10.1126/science.361.6404.742

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Huntington disease is a brutal brain malady caused by a mutant protein that inexorably robs victims of control of their movements and their minds. The slow decline, typically beginning in midlife, is agonizing for patients and families alike, not least because each child of an affected person has a 50% chance of inheriting the fatal disease. Now, for the first time, a drug being tested in humans has been shown to suppress the toxic protein. Michelle Dardengo of Coquitlam, Canada, was the first patient in the first human trial of that drug—a synthetic snippet of DNA developed by the biotechnology company Ionis Pharmaceuticals and recently licensed by Roche. The Swiss drug giant is preparing to launch a pivotal human trial in hundreds of patients. The first trial indicated that the drug is safe. But for Dardengo and untold others with the disease, the question that matters has yet to be answered: Does it slow or stop the disease?