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
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease
Our friends at @hdsa have produced this really good Q&A document about the latest huntingtin-lowering news.
Updates from day 3 of the Huntington’s Disease Therapeutics Conference: huntingtin protein - and lowering it
A surprising new paper sheds light on the role of the HD gene early in development. Should we worry?
Amazing news from Ionis and Roche! HTTRx drug successfully lowers harmful huntingtin protein in spinal fluid
Ionis says its trial of HTTRx, intended to lower huntingtin protein, is fully recruited and plans to extend it