New Huntington’s disease drug identified as ‘safe’ in recent clinical trial

A recent clinical trial has demonstrated that the drug, IONIS-HTTRx, is safe for use in people with Huntington’s disease. An international team of researchers has conducted a randomized, double blind, Phase I-IIA clinical trial for the use of IONIS-HTTRx in the treatment of Huntington’s disease. The main aim of the paper, published in the New England Journal of Medicine, was to determine whether the drug was safe for use in people with Huntington’s disease. Utilization of the antisense oligonucleotide IONIS-HTTRx in preclinical models has demonstrated that the drug inhibits synthesis of the mutant huntingtin protein, which causes Huntington’s disease. From...