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The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
Latest News
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First participant dosed in pioneering neural stem cell trial for Huntington’s disease
A new clinical trial for HD is underway. The first participant has been dosed in REGEN4HD, a study testing whether transplanting neural stem cells is safe as a potential treatment for HD.
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Evolving Needs, Evolving Care: Adapting Psychological Support Across Different Stages of Huntington’s Disease
⏱️ 9 min read | Huntington’s disease affects more than movement: it also has a major impact on mental health. Researchers explore why psychological care matters and how it needs to be adapted across each stage of HD.
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Sealing the gap: a case for slower, smarter DNA repair
⏱️ 7 min read | Your body assembles DNA like a team of workers who set and bond a pattern of tiles. A recent study shows that one special assistant might slow somatic CAG expansion and delaying HD onset.
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HDBuzz 2025 Annual Report: Here’s what you made possible
⏱️7 min read | In 2025, you helped us reach 329,000 people with independent, unbiased HD research news. Our 2025 Annual Report is here. Take a look at what we built together, and consider helping us keep going.
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Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515
One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine whether the drug truly slows HD progression
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May 2026: This Month in Huntington’s Disease Research
⏱️8 min read | May 2026: 2 year data from the votoplam trial suggests disease progression may slow; AMT-130 heads to the UK for regulatory review; pigs reveal immune cells invading the HD brain; and new science zooms in on toxic huntingtin fragments.
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Fixing the Recipe: Lowering a Slice of Huntingtin
⏱️7 min read | In a recent paper, scientists targeted a small, harmful piece of the huntingtin message (HTT1a) in HD mice. This reduced toxic protein clumps and delayed genetic changes, more than targeting full length HTT.
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Tagging the Trash: Turnover of Toxic Huntingtin
⏱️ 5 min read | A recent study suggests that strengthening the brain’s own clean-up system could slow Huntington’s disease.
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Prilenia’s Application for European Approval of Pridopidine Withdrawn
⏱️ 5 min read | Prilenia’s application for European approval of pridopidine was quietly withdrawn 6 months ago, in November 2025, after the EMA had already recommended refusing the drug. Here’s what happened, and why you’re only hearing about it now.
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Intruders in the Brain: What a Pig Model Reveals about Immune Cells in HD
⏱️7 min read | A recent study in pigs suggests the HD brain could be under attack from the body’s own immune system. The results reveal that invading cytotoxic T cells might contribute to neurodegeneration in HD, suggesting a new therapeutic pathway.
HDBuzz Trial Tracker
Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
REGEN4HD, Phase 1 stem cell implantation study commences
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
