New HD drug could be ‘ground-breaking’

A breakthrough that is being hailed as the most important development in HD since the HD gene was identified back in 1993 has been announced by pharma companies Ionis and Roche. It involves the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, which demonstrated a reduction in mutant huntingtin in the nervous system. The aim of the trial was to test tolerance and the safety of the drug before moving to further development. While the breakthrough is certainly important, it is too early to know if the symptoms of HD improve as a result of the drug. The initial trial enrolled 46 people with early HD symptoms in Germany, Canada and the UK. Read more at https://en.hdbuzz.net