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2025: Year in Review
Latest News
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Break Up With Your CAGs: How Three Letters Could Change Huntington’s Disease
Scientists engineered stem cells with “interrupted” CAG repeats to break up the toxic stretch. This may stop expansion, and could improve problems in cells that model Huntington’s disease. This study suggests that DNA spelling can drive the disease.
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Moving into the Fast Lane: uniQure and the FDA Are on the Same Track for Accelerated Approval
In an update shared today, uniQure announced alignment with the US drug regulator on key criteria for accelerated approval of drugs for Huntington’s disease.
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Interim update from Vico Therapeutics on their CAG-targeting drug, VO659
Vico Therapeutics have shared interim data about their drug, VO659, which targets the CAG expansion that causes several genetic diseases, including #Huntingtonsdisease and #spinocerebellarataxia
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Going boldly: First person treated in Phase 1 clinical trial by Alnylam Pharmaceuticals
A new Phase 1 clinical trial for the huntingtin-lowering drug ALN-HTT02 was initiated this week with the first dose given. Read on to learn details about the trial and how it compares to other ongoing huntingtin-lowering trials in the clinic.
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Giving Thanks to the Huntington's Disease Family Community for Advancing Research
At this time of giving, we want to give thanks to you, the Huntington’s disease (HD) community, for all you have done for HD research, bringing us closer to medicines which will one day slow or halt HD.
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Self-determination on the HD journey: the role of Advanced Care Planning
Advanced Care Planning is a tool that can allow people living with Huntington’s disease to compassionately engage with loved ones about their future care, taking thoughtful steps at their own pace while they’re able, to ensure their wishes are met.
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2024 HDBuzz Prize: Beyond nerve cells – who are the other players in the HD brain?
We're proud to announce Jenny Lange as a 2024 HDBuzz Prize winner! Our brains are made up of many types of cells that play different roles in the changes caused by HD. This study dives into a star-shaped cell called astrocytes and their role in HD.
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Face-to-face: Huntington’s disease families are heard by the FDA
The HDSA recently coordinated a meeting between Huntington’s disease families and the US regulatory agency that approves medicines, to amplify the community’s voice and help move us toward treatments.
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The dust has settled: Sage’s dalzanemdor won’t advance for cognitive impairment
Changes in thinking, learning, and memory are some of the most distressing and impactful in Huntington’s disease. Sage Therapeutics was advancing their drug dalzanemdor to treat these symptoms, but unfortunately this program will be halted.
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We are not alone, and are seeing more RED
Scientists have found more people have the genetic changes that underlie repeat expansion diseases, like HD, than previously thought. This new data tells doctors to consider expansion diseases more when diagnosing people with neurological symptoms.
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Huntington Study Group (HSG) Conference 2024 – Day 3
Read updates from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2024 Huntington Study Group conference.
