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Controlling DNA Scanning Machines Slow Expansion of CAG Repeats
A team of scientists have discovered small molecules that block the DNA repair protein MSH3, thought to be a key driver of repeat expansion in Huntington’s disease (HD). Although still at an early stage, this work opens the door to a new kind of therapeutic strategy: slowing down HD before...Read more
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2025 HDBuzz Prize: When Good Huntingtin Goes Missing: A Step Toward Designing Safe HD Treatments
A new study has shed light on the role of the regular huntingtin protein in the brain. For years, researchers have known that the faulty expanded huntingtin protein drives Huntington’s disease (HD), but this new study shows why the regular version matters for brain health. By lowering regular huntingtin (HTT)...Read more
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2025 HDBuzz Prize: On the Frontlines: Trusting the Hall Monitors of the Cell
Think about the last time you were stressed. What did you do to feel better? For many of us, it’s talking through our frustrations. Getting rid of things that stress out our cells also requires good communication. The central communicators? Chaperone proteins. Chaperone proteins are just like the chaperones at a...Read more
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September 2025: This Month in Huntington’s Disease Research
This month brought landmark therapeutic news, advances in biomarkers, deeper insights into what drives Huntington’s disease (HD), and fresh perspectives on mental health in HD. In particular among the developments, we learned from uniQure that the AMT-130 gene therapy appears to be slowing disease progression in their trial. Across the...Read more
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial
UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the key results from a study that is released quickly after data becomes available to the company...Read more
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2025 HDBuzz Prize: When the Repair Crew Breaks Down: How Expanded Huntingtin Disrupts DNA Repair
Every cell in our body is constantly fixing DNA damage that happens throughout our lifetime. Like a city sending out crews to mend roads and power lines, our cells rely on specialized proteins to keep our genetic code in a state of good repair. The huntingtin (HTT) protein has...Read more
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SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
On September 17, 2025, we received an encouraging update from Skyhawk Therapeutics’ Phase 1 study, taking place in Australia. The recent update suggests that SKY-0515 can lower the huntingtin protein as well as PMS1, another protein which is thought might also drive Huntington’s disease (HD). Importantly, the drug...Read more
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2025 HDBuzz Prize: MR-“why” am I feeling this way? How MRI is helping us understand why people with HD may sometimes be unaware of their symptoms
People living with Huntington’s disease (HD) experience changes in their thinking, memory, and behaviours. This is anarea of HD that is widely studied and fairly well understood. However, sometimes people with HD don’t realize these changes are happening. This is actually a symptom known as anosognosia. This symptom isn’t the same as being in denial, where a person avoids reality while still being aware of...Read more
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PROOF in the Publishing: Slicing the Data on Pridopidine
Pridopidine has been under investigation as a treatment for Huntington’s disease (HD) since the early 2000s. The most recent Phase 3 trial testing to see if pridopidine might improve signs and symptoms of HD, a study called PROOF-HD, did not meet its key goals. Furthermore, this summer...Read more
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2025 HDBuzz Prize: ACTing on HD: Exploring Acceptance and Commitment Therapy to Improve Mental Health in People with Huntington’s Disease and Their Caregiver
A group of researchers at the University of Pennsylvania, USA, has recently carried out an investigation to explore whether using a type of talking therapy, called Acceptance and Commitment Therapy (ACT), can help improve the mental wellbeing of people affected by Huntington’s disease (HD). The study, published in...Read more
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2025 HDBuzz Prize: Calm Before the Storm: Early Clues of Huntington’s Disease Found in Brainwaves
We know that HD affects how we move, think, and feel but these symptoms don’t appear overnight. Tiny hidden changes in the brain can begin years before a diagnosis. One way that scientists can detect these early shifts is by measuring brainwaves. This is a painless, non-invasive way...Read more
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Falling Into Hope: HDBuzz Fundraising Campaign 2025
This Fall, HDBuzz is proud to launch our “Falling Into Hope” fundraising campaign. Our goal is simple but ambitious: raise $30,000 in the next 8 weeks, by October 28. 2025 will be a landmark year in HD research. We’re closer than ever to disease-modifying therapies,...Read more
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August 2025: This Month In Huntington’s Disease Research
This month’s Huntington’s disease (HD) research roundup spans work from the dinner table to DNA repair. Scientists explored whether eating on a schedule could help clear toxic proteins, uncovered early signs of muscle loss in HD, and examined how childhood experiences shape adult mental health. Other teams investigated...Read more
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When DNA Repair Goes Off Script: How a Small Change in FAN1 Can Accelerate Huntington’s Disease
Two research teams have uncovered how a small change in FAN1, a DNA repair protein, can speed up Huntington’s disease (HD). In back-to-back papers in Nature Communications, they show how a single mutation known to influence when symptoms begin appears to prevent FAN1 from working properly. This ...Read more
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