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EHDN 2026 Part 1: What’s next in the Huntington’s disease treatment pipeline?
9 min read
Latest News
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
6 min read
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
11 min read
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
9 min read
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
9 min read
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
6 min read
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
6 min read
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
8 min read
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
7 min read
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.
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A sprinkling of good news for the treatment of HD chorea
2 min read
A new form of the chorea drug valbenazine (INGREZZA) has been approved by the United States Food and Drug Administration (FDA) for those who have difficulty swallowing pills.
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A new era for HDBuzz
12 min read
For over a decade HDBuzz has reported unbiased news about Huntington’s disease research and trials. As our beloved Ed and Jeff step back, Rachel, Sarah, and Leora look forward to upholding the HDBuzz mission.
HDBuzz Trial Tracker
FDA accepts BLA for Priority Review, with a target decision date of 1 May 2027.
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Updates from the ongoing Phase 3 INVEST-HD trial of votoplam
Updates from the Phase 1b/2a REGEN4HD trial of neural stem cell therapy hNSC-01
Updates from the ongoing Phase 2/3 FALCON-HD trial of SKY-0515
