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.

Tuesday, December 09, 2025

Huntington’s disease community urges FDA to get on the right side of history for uniQure gene therapy

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  Even as uniQure , in a December 4 press release , has reconfirmed – based on meeting minutes – the decision by the U.S. F...
Saturday, November 15, 2025

As uniQure seeks to overcome ‘dysfunction’ at the FDA, Huntington’s disease community rallies in defense of gene therapy drug

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As uniQure  seeks to overcome a decision by the U.S. Food and Drug Administration (FDA) to roll back its consultations regarding the firm’s ...
Wednesday, November 05, 2025

Understanding the FDA’s surprising step back on uniQure drug that slows Huntington’s disease

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  Less than six weeks after uniQure  announced that its gene therapy drug slowed the progression of Huntington’s disease b...
Friday, October 17, 2025

As hopes build about the first effective therapy, a Huntington’s disease family reunites

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  After 17 years of estrangement because of disagreements over family caregiving, in July I visited my hometown in Lake Cou...
Wednesday, September 24, 2025

Wonderful news: uniQure’s one-and-done drug slows Huntington’s disease

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  AMT-130 , a one-time gene therapy developed by uniQure , has successfully slowed the progression of Huntington’s disease....
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