No, not a puppy or a kitten or even a pet turtle, but a PET tracer to visualize huntingtin (HTT) protein in living subjects. PET stands for Positron Emission Tomography, an imaging technique that can ...
On June 30th, Skyhawk Therapeutics shared a press release with new twelve-month data from the ongoing Phase 1/2 trial of ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 small study from Brazil tested whether stem cells from human dental pulp, the soft tissue inside teeth, could help people with Huntington’s disease (HD). The results hint at small improvements on ...
CRISPR is short for “clustered regularly interspaced short palindromic repeats” – quite a mouthful! That’s essentially just science-speak for short strings of DNA letters that break up repeating parts ...
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 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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results