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.
In a surprising twist, oral HTT-lowering drugs also slow somatic expansion in the HTT gene. A new study that used cells in a dish for this fortuitous discovery identified the gene PMS1 as a key player in the slowing of CAG expansions.