The VIBRANT-HD study began in early 2022 and was a long-awaited trial of a huntingtin-lowering drug, branaplam, that could be taken by mouth. On Monday, August 8th, we learned that dosing has been ...
On June 30th, Skyhawk Therapeutics shared a press release with new twelve-month data from the ongoing Phase 1/2 trial of ...
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 ...
In plain language. Written by scientists. For the global HD community.
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.
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 ...
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 ...
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 ...
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 ...
The SIGNAL clinical trial was designed to test a drug called pepinemab in people with early Huntington’s disease. The key results of that trial were recently announced, and unfortunately, pepinemab ...
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 ...
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