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Huntington’s breakthrough may stop disease

11 December 2017

The first drug targeting the cause of Huntington’s disease was safe and well-tolerated in its first human trial, and successfully lowered the level of the harmful Huntingtin protein in the nervous system.

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After over a decade in pre-clinical development, this first human trial of huntingtin-lowering drug began in late 2015, led by Professor Sarah Tabrizi (UCL Institute of Neurology and UCLH consultant neurologist). The trial has been sponsored by Ionis Pharmaceuticals.

The trial involved enrolling 46 patients with early Huntington’s disease at 9 study centres in the UK, Germany and Canada. The UK centre was UCLH’s Leonard Wolfson Experimental Neurology Centre in the National Hospital for Neurology and Neurosurgery at Queen Square.

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