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, ...
A new study led by researchers from University College London has helped uncover some of the earliest changes that happen in people with the gene for Huntington’s disease (HD), long before obvious ...
People who develop Huntington’s disease (HD) are born with the genetic change that causes the disease. So why does it take decades, usually around 40 to 50 years, for the symptoms of the disease to ...
While HD primarily affects the brain, people with HD have subtle changes to their nervous system that can also affect heart rate and blood pressure. The scientists who worked on this paper had ...
A new CRISPR-based technology, called RIDE, is a leap forward for this trail-blazing technology. With the precision of a scalpel sharp enough to rewrite the very code of life, researchers have used it ...
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 ...
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 ...
While the genetic change that causes Huntington’s disease (HD) leads to several problems for cells, researchers believe they could stem from one core issue: the length of the genetic change increasing ...
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 ...
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 ...