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HDBuzz 2025 Annual Report: Here’s what you made possible
Latest News
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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Prilenia’s Application for European Approval of Pridopidine Withdrawn
⏱️ 5 min read | Prilenia’s application for European approval of pridopidine was quietly withdrawn 6 months ago, in November 2025, after the EMA had already recommended refusing the drug.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.
REGEN4HD, Phase 1 stem cell implantation study commences
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
