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HD2026 Milton Wexler Biennial Symposium Day 3
Latest News
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HD2026 Milton Wexler Biennial Symposium Day 2
⏱️ 23 min read | Day 2 of #HD2026 brought us updates on the mechanics of somatic instability, new ways to track HD in people, AI tools to help drive research forward, and developments in our understanding of the HTT protein. Catch up right here.
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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Prilenia’s Application for European Approval of Pridopidine Withdrawn
⏱️ 5 min read | Prilenia’s application for European approval of pridopidine was quietly withdrawn 6 months ago, in November 2025, after the EMA had already recommended refusing the drug.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.
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The Great Care Conundrum: What “Good” Care Looks Like in Huntington’s Disease
⏱️8 min read | What does “good care” look like in Huntington’s Disease? A new Dutch study asked familial and professional caregivers, and uncovered how differently it’s experienced in practice.
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
⏱️ 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here’s what that means, and what might be to come.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
⏱️10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.
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April 2026: This Month in Huntington’s Disease Research
⏱️ 8 min read | April 2026 showed us a toxic HTT fragment is center stage in mouse research, CRISPR is showing promise in mouse studies, AI and wearables move closer to the clinic, and 2 studies shine a light on the weight carried by HD caregivers.
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
