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AMT-130 at Four Years: Hopeful Signs, Complex Controls
12 min read
Latest News
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September 2026: This month in Huntington’s disease research
5 min read
September brought progress across the HD research landscape. From brain waves and visual thinking to DNA repeats, nutrition, inclusive care, new technologies, and encouraging SKY-0515 data, we round up a busy month in Huntington’s disease research.
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Screening the entire genome for new drug targets for HD
6 min read
Scientists screen the ENTIRE genome to find new potential therapeutic targets for HD. This ambitious study provides a wealth of data for HD researchers
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The third dimension: using minibrains to understand brain development changes in HD
10 min read
Researchers show that highly expanded CAGs in the HD gene can cause early developmental changes using 3D brain models called organoids. What’d they find?
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Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement
10 min read
Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?
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Could molecular handcuffs lower the protein that causes Huntington's disease?
6 min read
Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
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Exciting new Huntingtin lowering tool described
8 min read
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
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Details emerge of first Huntington’s disease gene therapy clinical trial
8 min read
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.
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Fresh Updates from First Huntingtin Lowering Study Publication
12 min read
Hot off the presses – New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
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An Updated Trial Design for Roche's Huntingtin Lowering Trial
8 min read
Roche announces an update to the GENERATION HD1 trial after reviewing the ASO open label extension study
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When interrupting is good: genetic hiccups that protect against Huntington's disease
12 min read
Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?
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Huntington's disease therapeutics conference 2019 – Day 3
12 min read
New insights into the huntingtin protein, and further progress in turning genetic quirks that modify the course of HD into treatments, on the final day of the therapeutics conference
HDBuzz Trial Tracker
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Updates from the ongoing Phase 3 INVEST-HD trial of votoplam
Updates from the Phase 1b/2a REGEN4HD trial of neural stem cell therapy hNSC-01
Updates from the ongoing Phase 2/3 FALCON-HD trial of SKY-0515
