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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
Latest News
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
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
⏱️ 8 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
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
⏱️10 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
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
⏱️ 7 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.
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January 2026: This Month in Huntington’s Disease Research
⏱️ 9 min read | January 2026 HD research recap: SKY-0515 shows promising safety data, community advocacy helps secure critical FDA meeting for AMT-130, plus new insights on sleep, vaccines, and symptom management. Read the full roundup!
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Phase 1 results announced for Skyhawk’s drug SKY-0515
Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with 60% reduction of huntingtin in blood at the highest dose tested. This data is an encouraging step as larger trials continue.
HDBuzz Trial Tracker
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
Additional insights from Phase 2 study results and update on plans for Phase 3 trial for votoplam
