Huntington’s disease breakthrough in first-in-human trial

Written by Roisin Conneely & Lauren Pulling, Editor

Results from a first-in-human trial led by researchers at University College London (UCL; UK) have been hailed as “of ground breaking importance for Huntington’s disease patients and their families”. For the first time, researchers have successfully lowered levels of mutant huntingtin protein in patients in a trial of Ionis-HTTRx, an antisense oligonucleotide developed by Ionis Pharmaceuticals in partnership with Roche. Scroll down to hear from Edward Wild, a senior researcher at the Huntington’s Disease Centre at UCL, and researcher on this trial, who spoke to Neuro Central about the trial findings The Phase I/II trial enrolled 46 patients across nine...

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