More good news for the HD community from uniQure, whose one-and-done gene therapy appears safe and shows hints of slowing down signs and symptoms of the disease
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 4 of the 2024 HDF Milton Wexler Biennial Symposium #HD2024
Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
Read updates from clinical trials and scientific research on Huntington’s disease from Day 2 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
Sage Therapeutics announced results from SURVEYOR, a trial that looked at cognitive changes in people with HD, and tested short-term safety of dalzanemdor. The study was small but met key goals, and additional trials are in progress.
New work from two groups advances tools to model the blood-brain barrier and could improve the way drugs are delivered for brain diseases. This builds on our ability to study, and eventually treat, Huntington’s disease.