We know that lowering the level of mutant huntingtin protein in HD mouse models significantly improves symptoms reminiscent of HD, providing hope that similar treatments in people may be effective. (en.hdbuzz.net)
Clumps of mutant huntingtin protein in brain cells are a hallmark of HD, and they build up slowly, occupying more and more cells over time. (en.hdbuzz.net)
The phase 1 / 2a study of IONIS - HTTRx was recently completed and showed the therapy to be safe and well - tolerated but also showed it's ability to reduce the level of mutant huntingtin in a dose dependent manner in Huntington's Disease patients. (ehdn.org)