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When the Brainâs Orchestra Falls Out of Tune: A New Map of Huntingtonâs Disease Progression
Latest News
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Details emerge of first Huntingtonâs disease gene therapy clinical trial
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a âsingle-shotâ virus delivery system.
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Fresh Updates from First Huntingtin Lowering Study Publication
Hot off the presses – New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
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An Updated Trial Design for Roche's Huntingtin Lowering Trial
Roche announces an update to the GENERATION HD1 trial after reviewing the ASO open label extension study
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When interrupting is good: genetic hiccups that protect against Huntington's disease
Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?
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Huntington's disease therapeutics conference 2019 – Day 3
New insights into the huntingtin protein, and further progress in turning genetic quirks that modify the course of HD into treatments, on the final day of the therapeutics conference
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Huntington's disease therapeutics conference 2019 – Day 2
New tools to bridge the gap between the lab and patients in our update from day 2 of the 2019 HD Therapeutics conference
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Huntington's disease therapeutics conference 2019 – Day 1
HDBuzz reports from the annual Huntingtonâs disease therapeutics conference in Palm Springs
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Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants âInvestigational New Drugâ status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntingtonâs 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)