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Huntington’s disease treated successfully for first time in UK gene therapy trial | Surgical procedure to treat devastating illness slowed progress of disease by 75% in patients after three years
Surgical procedure to treat devastating illness slowed progress of disease by 75% in patients after three years
Perry, a support worker from Liverpool, now has mild motor symptoms and hopes she may benefit from gene therapy in future, saying that she and friends in the Huntington’s community shared tears of joy on learning of the trial results. Three years after treatment, those on the high dose of the drug had an average of 75% slowing of the progression of the disease, based on tests of motor function, cognition and the patients’ experience of day-to-day life. “We are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington’s disease,” said Walid Abi-Saab, the chief medical officer of uniQure, adding that the company hoped to submit the drug for approval in the US early next year.
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