HDBuzz-Aggregator

One mystery that many scientists think holds the key to curing HD is its mysterious age of onset. Although people with HD carry the expanded gene from birth, they generally don’t develop symptoms until later in life, suggesting something bad is brewing beneath the surface! One explanation, which has gained...Read more

Ever since large genetic studies in Huntington’s disease (HD) revealed that the longer the CAG expansion, the earlier symptoms appear, we’ve known that repeat length matters. Recent work has highlighted just how that repeat length increases within vulnerable brain cells — from about 50 CAGs to over a thousand.  ...Read more

On December 4, 2025, uniQure announced they have received the final meeting minutes from their October 29 pre-Biologics License Application (BLA) meeting with the FDA regarding AMT-130. The minutes confirm what was reported in early November: the FDA currently believes the Phase I/II data are unlikely...Read more

When it comes to thinking about the effects of Huntington’s disease (HD), most people automatically start to think about the brain due to the severe symptoms caused by the breakdown of brain cells. It’s easy to forget that the gene which causes HD is present throughout the whole body –...Read more

Depression and anxiety are common symptoms of Huntington’s disease (HD), and they can make everything harder. New research explores how sertraline, a widely-used antidepressant, affects protein production in HD cells and mice, finding that it prevents motor problems in HD mice and is linked to slower functional decline in people...Read more

Welcome back to the HDBuzz monthly research roundup! November was a busy month, with new developments in everything from gene therapy and stem cells to DNA repair, genetic modifiers, and protein folding. Here’s a friendly guide to what scientists learned this month, why it matters, and what it might mean...Read more

Imagine a busy clothing factory. Proteins are like newly sewn shirts coming off the machines. They’re floppy, wrinkled, and can create a giant mess if they’re left unfolded. Normally, the cellular clothing factory employs “chaperone proteins” that act like skilled pressers. They grab each shirt, apply energy from a hot...Read more

SOM3355 is an investigational therapy aimed at managing multiple symptoms of Huntington’s disease (HD) and recently crossed two key regulatory milestones. In September, the European Medicines Agency (EMA) issued a positive opinion supporting orphan drug designation for SOM3355. Now, following a productive End-of-Phase-2 meeting in the United States, the US...Read more

Scientists often use genetics, the study of DNA, to understand the cellular changes that cause disease. By comparing people’s DNA with their symptoms, they can pinpoint specific genetic differences, called variants, that influence the severity of a disease. Huntington’s disease (HD) is well-suited for genetic analysis because of its well-understood...Read more

Our bodies are experts at looking after our DNA and are continuously monitoring for any damage that needs to be repaired to keep us healthy. Parts of DNA that are very repetitive, like the sequence causing Huntington’s disease (HD), are very tricky to look after and our body can try...Read more

A small study from Brazil tested whether stem cells from human dental pulp, the soft tissue inside teeth, could help people with Huntington’s disease (HD). The results hint at small improvements on some movement measures, but the study had few participants who were tracked over a short timeframe, and many...Read more

Why do some people with intermediate CAG repeats, a genetic “gray zone” in Huntington’s disease, develop neurological symptoms while others do not? This article covers a recent study that tackled this question by looking for somatic expansion, which is a tiny changes in DNA, in individuals across various CAG repeat...Read more

In a very brief press release from uniQure on November 3, 2025 we learned that the company is no longer aligned with the FDA on moving their drug AMT-130 forward. Just a few weeks ago, positive news from their ongoing clinical trials took the world by storm. Their...Read more

This month brought new revelations across biomarkers, basic science and community gathering. In particular, we saw how DNA-repair targeting strategies are moving into focus, how gaps in diagnosis are being quantified, and how the annual Huntington’s Disease Clinical Research Congress 2025 deepened connections across the field. Together these developments reflect...Read more

A new study in mouse models reveals how Huntington’s disease (HD) disrupts brain development over time, even long before symptoms appear. Using advanced sequencing tools and spatial transcriptomics, a technique that maps where in the brain genes are activated, researchers discovered early warning signs that could help explain why some...Read more

The past 4 weeks have been a whirlwind in the Huntington’s disease (HD) community. On September 25th we had an update from uniQure about a drug they’re testing for HD in ongoing clinical trials. The news was positive and it took the world by storm, producing jaw dropping headlines...Read more

Huntington’s disease (HD) is caused by repeating C-A-G letters of genetic code that are too long. Everyone who develops HD is born with 36 or more CAG repeats, but not everyone with 36 or more CAG repeats is actually diagnosed with HD. That’s because either they are not old enough...Read more

Welcome back for the final day of the Huntington’s Disease (HD) Clinical Research Congress in Nashville, Tennessee! Translational issues in HD The first session will focussed on translational issues in HD – how we get research to people...Read more

We’re back for Day 2 of Huntington’s Disease (HD) Clinical Research Congress in Nashville, Tennessee! Daniel Claassen from HSG and Cristina Sampaio from CHDI kick off the meeting with an overview of what we’ll hear about in HD clinical research and discovery.  They are highlighting the search...Read more

This month, HDBuzz is attending the first Huntington’s Disease (HD) Clinical Research Congress in Nashville, Tennessee. Gathered at this meeting are hundreds of scientists, doctors, and industry representatives who have come together to talk about HD clinical research and care. This conference has been organized by the Huntington Study Group...Read more

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

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

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

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

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

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

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

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

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