Latest News
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Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
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Advances on many fronts in the battle against the protein that causes Huntington's disease
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
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HDSA FAQ on the Roche/Genentech RG6042 program
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
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Kids sometimes get Huntington’s disease too
Finally, a big study that shows what childhood HD looks like. This will help us work out if new drugs work in children too
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Roche announces details of its 'pivotal' huntingtin-lowering study
The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease
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Disappointing news from LEGATO-HD trial of laquinimod in Huntington's disease
The LEGATO-HD trial of laquinimod did not slow progression of Huntington's disease. Here's the lowdown.
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HDBuzz Enrolls in Enroll-HD
As promised, HDBuzz's Jeff Carroll finally signed up for the Enroll-HD study. Learn more about this important global HD "observational study" here.
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First Dolly, now Piglet; a new Huntington’s disease pig knock-in model confirmed
For the first time in HD research, an animal model has been created in pigs using 2 cutting edge DNA editing t
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New collaboration seeks to speed Huntington's disease drug licensing
Critical Path Institute launches new initiative to get HD drugs licensed as quickly as possible
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HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche
Our friends at @hdsa have produced this really good Q&A document about the latest huntingtin-lowering news.
HDBuzz Trial Tracker
Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
Updates from the ongoing placebo-controlled Phase 2/3 FALCON-HD trial
Previously featured
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March 2026: This Month in Huntington’s Disease Research
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Two Heads Are Better Than One: Combined Physical and Music Therapy for Late-Stage Huntington’s Disease
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A Guest Perspective for Gratitude Day: Why Huntington’s Disease May Be Neuroscience’s Best Investment
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The Girl at the End of the World: A Gratitude Day Reflection on the Place that Built Our Science
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Turning down mismatch repair genes slows Huntington’s repeat growth in human neurons
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