At Risk for Huntington's Disease

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

Wednesday, April 22, 2026

As FDA faces demands for greater clarity, uniQure presses ahead in search for Huntington’s disease gene therapy approval

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   As the reportedly dysfunctional U.S. Food and Drug Administration (FDA) faces demands for greater clarity in the wake of r...
Sunday, April 05, 2026

A Senate probe of the FDA and a letter from advocates to Trump could aid in approval of Huntington’s disease gene therapy

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   Two key political developments could aid uniQure’s  effort to seek approval of its historically efficacious Huntington’s d...
Monday, March 09, 2026

CHDI chief scientist: ‘at the precipice’ of treatments, Huntington’s disease community must ‘persevere’ through the stress

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   As researchers have hypothesized that lowering (reducing) the amount of abnormal huntingtin protein in the brains of people...
Monday, March 02, 2026

FDA recommends a full clinical trial for uniQure Huntington’s disease drug, further delaying potential approval

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   Delaying but not blocking AMT-130, uniQure’s  emerging gene therapy to slow Huntington’s disease progression, the U.S. Food...
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