FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
Huntington's disease research news. In plain language. Written by scientists. For the global HD community.
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
Finally, a big study that shows what childhood HD looks like. This will help us work out if new drugs work in children too
The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease
The LEGATO-HD trial of laquinimod did not slow progression of Huntington's disease. Here's the lowdown.
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
Our friends at @hdsa have produced this really good Q&A document about the latest huntingtin-lowering news.