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Prilenia shares HD trial data with CHDI to help future research
Latest News
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HD2026 Milton Wexler Biennial Symposium Day 1
⏱️ 28 min read | Day1 of HD2026 is a wrap! From what human brains can teach us about HD, to HTT1a, somatic CAG expansion and the normal biology of HTT, today’s talks spanned the spectrum from fundamental biology to potential treatments.
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Turning down mismatch repair genes slows Huntington’s repeat growth in human neurons
⏱️ 10 min read | In a human cellular system, scientists dialed back mismatch repair genes to reduce CAG expansion by up to 69%. This work is in its early days, but it shows promise for “anti-expansion” therapies that could be used to delay HD onset.
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Huntington’s Disease Therapeutics Conference 2026 – Day 3
⏱️ 29 min read | Day 3 of the HD Therapeutics Conference dives into breakthrough science, new tools and bold ideas that are pushing HD research toward better treatments.
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Huntington’s Disease Therapeutics Conference 2026 – Day 2
⏱️ 18 min read | HDBuzz is back with Day 2 of the 21st annual CHDI HD Therapeutics Conference in Palm Springs, CA. We heard about new techniques to zoom in on HD biology in extraordinary detail.
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Huntington’s Disease Therapeutics Conference 2026 – Day 1
⏱️ 33 min read | HDBuzz is at CHDI’s Huntington’s Disease Therapeutics Conference, ready to bring you live updates from the most recent research in the field. Get ready for some exciting science!
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
⏱️ 8 min read | An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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Vico’s Trial Adjusts With Twice-Yearly Dosing, And The U.S. Is Next
⏱️ 6 min read | Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.
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February 2026: This Month in Huntington’s Disease Research
⏱️ 8 min read | February 2026 HD research recap: published trial data from branaplam trial; new insights into the genetics of the HD “grey zone”; plus worms, brain circuits, eye tracking, and the hidden psychological weight of living at risk.
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A Window into the Eyes: Using Ocular Biomarkers to Track Progression in Huntington’s Disease
A new review from the University of Cambridge explores ocular biomarkers in HD, including retinal scans and eye movement tracking.
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A Fault In The Supercomputer
⏱️ 8 min read | Your brain is an advanced supercomputer. But in HD, a core processor starts to malfunction. A research group at UCLA has taken a close look to visualize the specific circuits going haywire – and how they look different in HD.
HDBuzz Trial Tracker
Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
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REGEN4HD, Phase 1 stem cell implantation study commences
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
