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EHDN 2026 Part 1: What’s next in the Huntington’s disease treatment pipeline?
9 min read
Latest News
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Skyhawk’s drug trial takes flight in the US
4 min read
Skyhawk’s FALCON-HD trial has dosed the first participants in the US with the drug, SKY-0515. The once-daily pill targets two drivers of HD, but does it actually slow disease? That’s the big question this study hopes to answer.
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FDA sets May 2027 decision date on AMT-130 gene therapy for HD
4 min read
The FDA has accepted uniQure’s application for AMT-130, an experimental gene therapy for Huntington’s disease, and set a May 2027 decision date. Here’s what this milestone means, and what still needs to happen.
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October 2026: This Month in Huntington’s Disease Research
4 min read
From sticky thoughts and pigs that model HD to DNA repair and promising drug trial results, October brought fresh insights into HD. Here’s what you might have missed!
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Turning down huntingtin: Alnylam’s brezosiran shows promising early result
6 min read
An experimental treatment for HD developed by Alnylam has shown long-lasting huntingtin lowering after just one dose, with encouraging early safety data. A pivotal trial is now planned for 2027 to figure out if this drug, brezosiran, can slow HD.
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Blurred Lines: When Similar Symptoms Can Mean Different Things
7 min read
When someone with Huntington’s disease experiences new symptoms, is HD always the cause? Not necessarily. A new review explores catatonia, an uncommon condition that can cause changes that overlap with HD symptoms.
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Picking MSH3’s Pocket: New Insights into CAG Repeat Expansion
8 min read
Scientists discovered a pocket on a protein called MSH3 that helps it grab DNA containing CAG repeats. By blocking this interaction, they slowed the growth of CAG repeats in cells, without removing the protein entirely.
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What Pigs Revealed that Mice Missed in Huntington’s Disease
9 min read
A change in one gene can disrupt thousands of others in HD. A study in pigs suggests an explanation: expanded huntingtin interferes with chemical marks on DNA. The same effect wasn’t seen in mice, showing that the animal model matters.
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Mending the fabric of your genes
6 min read
Cells constantly mend tears and rips in the fabric of your DNA, like a cherished pair of jeans. Sometimes this mending can add C-A-Gs by mistake. A group of scientists examined whether other kinds of rips in the genetic fabric may be occurring in HD.
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Could rewiring the brain’s “brakes” improve movement in Huntington’s disease?
6 min read
Scientists discovered that a specific type of brain cell becomes unusually quiet in Huntington’s disease. By restoring the activity of these cells in mice, they were able to rebalance brain circuits and improve movement.
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Your Voice Matters: Using Speech to Monitor Huntington’s Disease
9 min read
HD researchers developed an AI-powered tool that analyzes a short counting task. Its speech measures reflected clinical scores and HD-related changes over time, suggesting it could be used as a remote monitoring tool in future.
HDBuzz Trial Tracker
FDA accepts BLA for Priority Review, with a target decision date of 1 May 2027.
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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
