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A first for CRISPR gene editing could have wider applications for human disease
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?
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Scientists identify precisely how pridopidine works in models of Huntington’s disease
New studies pinpoint precisely how the drug pridopidine works in models of Huntington’s disease
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Huntington's disease therapeutics conference 2021 – Day 3
Catch up on all of the latest updates from day 3 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021
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Huntington’s disease therapeutics conference 2021 – Day 1
Catch up on all of the latest updates from day 1 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021 including presentations from Wave, Roche and Triplet.
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Huntington's disease therapeutics conference 2021 – Day 2
Catch up on all of the latest updates from day 2 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021
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Huntington’s disease clinical trial round up
Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease
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Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials
Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.
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Details emerge of first Huntington’s 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 ‘single-shot’ 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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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?