A small clinical trial of high-dose biotin and thiamine for treating HD is being planned in Spain. This trial is based on research that links HD to another neurodegenerative disease called biotin-thiamine responsive basal ganglia disease (BTBGD).
The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
Scientists recently used an antihistamine to quiet dopamine messages in the brain and treat HD-like symptoms in mice. But beware the hype suggesting that allergy medicines could be used to slow down 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
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.
New tools to bridge the gap between the lab and patients in our update from day 2 of the 2019 HD Therapeutics conference
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
For the first time in HD research, an animal model has been created in pigs using 2 cutting edge DNA editing t