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, ...
Usually, there are helper proteins, called “chaperones,” that work throughout the cell to make sure other proteins fold correctly and prevent them from clumping. However, in diseases like HD, this ...
For those who were following the live tweets from HDBuzz about the CHDI HD Therapeutics Conference or tuned in to the HDSA Convention, we may have caught your attention with the new HD staging system.
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 ...
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 ...
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, ...
Last month, we relayed positive news from uniQure’s trial testing AMT-130, a gene therapy delivered via brain surgery to lower huntingtin (HTT). Data released by uniQure in June suggested AMT-130 was ...
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 ...
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 ...
On May 5th, PTC Therapeutics released results from their ongoing Phase 2 PIVOT-HD clinical trial for PTC-518, now called votoplam. Excitingly, they announced that this trial met its primary endpoint – ...
The outcomes of the GENERATION HD1 trial have just been published in a scientific journal, nearly three years after the study was halted. In March of 2021, the HD community was hit with the difficult ...
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