-
EHDN 2026 Part 2: What’s the latest news for Huntington’s disease clinical trials?
12 min read
Latest News
-
EHDN 2026 Part 1: What’s next in the Huntington’s disease treatment pipeline?
9 min read
From stopping CAG repeats from growing to finding new ways to tackle harmful huntingtin, scientists at the EHDN Clinical Research Congress 2026 shared exciting ideas for the next generation of HD treatments. Here’s what’s coming down the pipeline.
-
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.
-
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.
-
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!
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
HDBuzz Trial Tracker
FDA accepts BLA for Priority Review, with a target decision date of 1 May 2027.
Show 8 more Show less
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
