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AMT-130 at Four Years: Hopeful Signs, Complex Controls
12 min read
Latest News
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September 2026: This month in Huntington’s disease research
5 min read
September brought progress across the HD research landscape. From brain waves and visual thinking to DNA repeats, nutrition, inclusive care, new technologies, and encouraging SKY-0515 data, we round up a busy month in Huntington’s disease research.
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
7 min read
An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
10 min read
The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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Vico’s Trial Adjusts With Twice-Yearly Dosing, And The U.S. Is Next
6 min read
Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.
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February 2026: This Month in Huntington’s Disease Research
7 min read
February 2026 HD research recap: published trial data from branaplam trial; new insights into the genetics of the HD “grey zone”; plus worms, brain circuits, eye tracking, and the hidden psychological weight of living at risk.
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A Window into the Eyes: Using Ocular Biomarkers to Track Progression in Huntington’s Disease
6 min read
A new review from the University of Cambridge explores ocular biomarkers in HD, including retinal scans and eye movement tracking.
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A Fault In The Supercomputer
8 min read
Your brain is an advanced supercomputer. But in HD, a core processor starts to malfunction. A research group at UCLA has taken a close look to visualize the specific circuits going haywire – and how they look different in HD.
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Split Between Two Worlds: The Psychological Challenges for People at Risk of Huntington’s Disease
7 min read
Living at risk for Huntington’s disease imposes a massive psychological burden. A small interview-style study of at risk 12 people dove into how these people cope day-to-day.
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Putting it in print: oral drug branaplam lowered huntingtin, but safety concerns halted development
9 min read
Results from the VIBRANT-HD trial are now published in a peer reviewed journal. This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial.
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A hitch in the stitch reveals why DNA sequence patterns matter in Huntington’s disease
9 min read
Two individuals with the same CAG repeat count can have vastly different HD journeys. A new study looking at individuals with 36-42 repeats shows that DNA sequence patterns play a major role in symptom onset and progression.
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Worms to the Rescue: Separating the Good, the Bad, and the Clumpy Huntingtin
6 min read
A team of researchers developed a simple platform to test how toxic different types of HTT protein clumps are using a laboratory worm model. They showed that certain types of clumps are harmful to the worms, while others are not.
HDBuzz Trial Tracker
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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
