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A gene therapy for Huntington’s disease made by the Dutch biotech firm Uniqure showed early promise in a preliminary study of a handful of patients, the company said Wednesday.

Evaluable data were available from only four patients who had received the treatment, code-named AMT-130, and three in a control group who had undergone a sham treatment. All were in the early stages of the fatal neurodegenerative disease.


Twelve months after treatment, the four treated patients saw levels of mHTT, the protein that causes Huntington’s, decline by between 44% and 71%, or 53.8% on average, in their cerebrospinal fluid. By comparison, the three patients who received the sham treatment saw levels of mHTT protein decrease by an average 16.8%, with individual results ranging from a 35% increase to a 47% decrease.

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