5 min read |The Huntington’s disease community’s unprecedented mobilization helped secure a high-priority FDA meeting to discuss the path forward for AMT-130.

Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with 60% reduction of huntingtin in blood at the highest dose tested. This data ...

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 ...

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.

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 ...

We tend to think of DNA as a fixed blueprint, an overarching plan for the biological bricks and bridges that constitute our cells, organs, and bodies. But like any good plan, DNA is actually dynamic ...

Scientists at Novartis and The Children’s Hospital of Philadelphia have recently published a paper detailing how the drug branaplam, originally developed for the neurological disease spinal muscular ...

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 ...

6 min read | Most people with HD report sleep problems, & new research suggests sleep quality is linked to many aspects of living with the disease. Wearables tracked people with HD for a year & ...

HDBuzz: 2024 was a big year for HD research, particularly in the clinical space. Which breakthroughs or advances in HD research from the past year are you particularly excited about? HDBuzz Editors-in ...

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, ...