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EAN 2021 | Huntington’s disease: developments on the horizon

Anne Rosser, PhD, FRCP, Cardiff University, Cardiff, UK, reviews the various therapeutic approaches under development for Huntington’s disease. A lot of progress has been made in a range of different strategies, including huntingtin-lowering therapies. Of these, only anti-sense oligonucleotides have reached clinical trials. Allele-specific products will be coming into trials soon, and new approaches for lowering mutant huntingtin will emerge. Different strategies involve targeting somatic expansions, meaning that DNA mismatch repair proteins might be an additional druggable target. Histone deacetylase inhibitors and cell-based therapies are also being investigated. In the future, it will be essential to understand how to combine different treatment strategies and at what disease stage to introduce each therapy. This interview took place during the European Academy of Neurology 2021 congress.