Dec 11, 2017

In early trial, new drug silences Huntington’s disease gene

An experimental drug could slow the spread of Huntington's disease, giving hopes to patients suffering from uncontrolled movements and mental confusion associated with the disease, reports The Guardian. An early-stage trial of the drug was conducted with 46 patients in the UK, Germany and Canada.

Why it matters: Huntington's disease is an inherited condition resulting from a genetic mutation. Current treatments can only help minimize the symptoms, instead of slowing it down.

Professor Sarah Tabriz at UK's University College of London said in a statement on Monday that the drug has lowered the level of the “toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”

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