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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
December 04, 2018
Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
January 30, 2019
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
March 05, 2019
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?
March 14, 2019
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
March 21, 2019
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
May 07, 2019
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.
August 05, 2019
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
November 12, 2019
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?
January 03, 2020
Screening the entire genome for new drug targets for HD
Scientists screen the ENTIRE genome to find new potential therapeutic targets for HD. This ambitious study provides a wealth of data for HD researchers
February 23, 2020
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