HDBuzz Aggregator

Huntington’s disease (HD) is caused by a repeated stretch of the genetic letters C-A-G within the huntingtin (HTT) gene above a critical number. If the repeats exceed 40, then signs and symptoms of HD will begin at some point in that person’s life, if they live long enough. The disease-causing...Read more

A new Huntington’s disease (HD) clinical trial has reached an important milestone: the first participants have now been dosed in POINT-HD, a Phase 1 study testing a new, selective huntingtin-lowering drug. RG6496 is designed to only lower levels of the expanded form of huntingtin (HTT), preserving levels of regular HTT....Read more

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