SKY-0515 is a daily oral drug designed to lower both huntingtin, the protein that drives HD, and PMS1, a DNA repair protein linked to somatic CAG repeat expansion. Researchers hope this dual-target ...
In a recent study, scientists created a chemical that breaks down expanded huntingtin HTT clumps in HD cells and mouse models. This chemical finds HTT clumps and targets them to be broken down or ...
Today, on June 17, 2026, uniQure shared a press release with an update from their recent Type B meeting with the FDA. In ...
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 ...
This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...
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 ...
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 ...
A 14-year study tracking NfL levels in people with the HD gene shows this tiny protein can signal disease progression many years before symptoms start. The longest follow-up study yet adds weight to ...
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 ...
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 ...
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.
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