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)
Emphasising their desire to move the drug forward, in a separate announcement made directly to the HD community, Ionis said «Upon study completion, the next step for this program will be to conduct a study to investigate if decreasing mutant huntingtin protein with IONIS - HTTRx can slow the progression of this terrible disease.» (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)