drug-development
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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
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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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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 Huntingtonās 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