Photo: Vidal Balielo Jr./Pexels
One of the most devastating diseases – Huntington's – has been successfully treated for the first time in a trial which involved 29 patients and used gene therapy given during 12 to 18 hours of delicate brain surgery. Three years after surgery, there was an average 75% slowing of the disease, giving patients decades of "good quality life", said Prof Sarah Tabrizi, director of the University College London Huntington's Disease Centre. Dr Walid Abi-Saab, chief medical officer at uniQure, said the treatment had "the potential to fundamentally transform" the disease. Results have been released in a statement by the company, but not yet published in full. UniQure says it will apply for a license in the US in the first quarter of 2026 with the aim of launching the drug later that year. Approximately 75,000 people have Huntington's disease in the UK, US and Europe.
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