By setting up both reactions side by side in a test tube, the team revealed a literal tug-of-war between two DNA-repair pathways acting on the same HTT repeat. Which side wins likely determines ...
UniQure received FDA’s meeting minutes on AMT-130. While there were no new updates, the community’s response has been ...
Studies in mice that model Huntington’s disease suggest that prebiotics – the food that feeds our gut bacteria – might ...
As 2025 winds down, HD research is more active, more interdisciplinary, and more globally connected than ever. Whether it’s genetic modifiers, new drug targets, or next-generation clinical trials, ...
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 ...
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 ...
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, ...
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 ...
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 ...
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 ...
A BLA, or Biologics Licensing Application, is the formal dossier of information that a company uses to request permission to market a biologic, which is a therapy created from living organisms, like ...
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 ...
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