Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
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.
Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
As promised, HDBuzz's Jeff Carroll finally signed up for the Enroll-HD study. Learn more about this important global HD "observational study" here.
For the first time in HD research, an animal model has been created in pigs using 2 cutting edge DNA editing t
Critical Path Institute launches new initiative to get HD drugs licensed as quickly as possible
Can we test drugs to delay or prevent the onset of Huntington's disease? New research suggests it's possible