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Unlocking the Mind’s Eye: How Huntington’s Disease Changes How We See and Process the World
Latest News
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Hereditary Disease Foundation (HDF) conference 2024 – Day 2
Read updates from clinical trials and scientific research on Huntington’s disease from Day 2 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
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Hereditary Disease Foundation (HDF) conference 2024 – Day 1
Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
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.
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Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial
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
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Positive news from Wave Life Sciences SELECT-HD trial
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
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No pivot needed for PTC-518
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.
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SURVEYOR opens the door for drugs that treat cognition
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.
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The VIP section: velvet ropes for the brain and how to get in
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.
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Two birds, one stone: HTT-lowering drugs also target CAG expansions
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.
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Hats off to brain donors on Brain Donation Awareness Day
May 7 is Brain Donation Awareness Day. Today we highlight the selfless donation that many HD families have made, sending our gratitude, sharing research updates made with those precious brains, and detailing resources for brain donation.