Therapies in the pipeline for Huntington's disease

Anne Rosser, PhD, FRCP, Cardiff University, Cardiff, UK, gives an overview of the progress made in Huntington’s disease research in the last 20 years. Currently, there are a large number of potential treatments that are gradually progressing towards the clinic, with some approaches already being evaluated in clinical trials, primarily huntingtin-lowering strategies. The PROOF-HD trial (NCT04556656) is a Phase III study evaluating the efficacy and safety of pridopidine in patients with early-stage of Huntington’s disease. Additionally, there has been a lot of progress in targeting somatic expansion. Another potential strategy for the treatment of Huntington’s disease currently under investigation is cell-based therapies. This interview took place during the European Academy of Neurology 2021 congress.