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Tooth fairy therapy? Stem cells from teeth tested in a small Huntington’s disease trial
Latest News
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Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement
Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?
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Could molecular handcuffs lower the protein that causes Huntington's disease?
Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
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Exciting new Huntingtin lowering tool described
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
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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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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 Huntington’s disease therapeutics conference in Palm Springs