drug-development
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When genes are unstable: targeting somatic instability in HD
CAG repeats expand in some parts of the body and brain as people with HD get older, a phenomenon known as somatic instability. Learn more about how researchers are exploring somatic instability and DNA repair to design therapies for HD.
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HD Young Adult Study defines the sweet spot: symptom-free with measurable changes
We know that HD-related changes can occur many years before symptom onset, but how early do those changes begin? A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults.
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What does COVID-19 mean for Huntingtonās disease families and HD research?
COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?
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New molecule can reverse the Huntington's disease mutation in lab models
A collaborative team of scientists from Canada and Japan have identified a small molecule which can change the CAG-repeat length in different lab models of Huntington's disease. #HuntingtonsDisease #DrugDiscovery
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Huntingtonās disease therapeutics conference 2020 – Day 1
HDBuzz reports from the annual Huntingtonās disease therapeutics conference in Palm Springs
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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 ā 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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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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Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants āInvestigational New Drugā status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntingtonās Disease patients