Sarah J Tabrizi, Blair R Leavitt, G Bernhard Landwehrmeyer, Edward J Wild, Carsten Saft, Roger A Barker, Nick F Blair, David Craufurd, Josef Priller, Hugh Rickards, Anne Rosser, Holly B Kordasiewicz, Christian Czech, Eric E Swayze, Daniel A Norris, Tiffany Baumann, Irene Gerlach, Scott A Schobel, Erika Paz, Anne V Smith, C Frank Bennett, Roger M Lane
BACKGROUND: Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT , resulting in a mutant huntingtin protein. IONIS-HTTRx (hereafter, HTTRx ) is an antisense oligonucleotide designed to inhibit HTT messenger RNA and thereby reduce concentrations of mutant huntingtin. METHODS: We conducted a randomized, double-blind, multiple-ascending-dose, phase 1-2a trial involving adults with early Huntington's disease...
May 6, 2019: New England Journal of Medicine