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EHDN 2022 | Moving forwards after disappointing data in the Huntington’s disease space

Recent years have seen disappointment in the Huntington’s disease (HD) therapeutics space, with several high-profile clinical trials terminated or suspended. The Phase III GENERATION HD1 study of tominersen was stopped after it was seen that the drug was not having any benefit, and those receiving the most frequent dosing were actually performing worse. The Phase I/II PRECISION-HD trials of WVE-120101 and WVE-120102 were also discontinued due to lack of efficacy. Most recently, the VIBRANT-HD trial of branaplam was temporarily suspended due to safety concerns surrounding peripheral neuropathy. Patrick Weydt, MD, PhD, University of Bonn Medical Center, Bonn, Germany, shares his thoughts on how the HD field should move forward after these unexpected outcomes. He notes that while the trials did not give the results investigators had hoped for, they still provide information that is helping to advance our understanding of disease and how trial design and therapeutic agents should be adjusted for the future. This interview took place during the European Huntington’s Disease Network 2022 Plenary Meeting.

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