UK researchers announced a breakthrough gene therapy that slowed Huntington's disease for the first time. Early-stage trials at University College London showed some patients’ condition progressed 75% slower over three years. The therapy, AMT-130, is delivered via direct brain injection.

London

UK scientists have reported a major advance in the treatment of Huntington's disease, a rare inherited brain disorder. A recent study found that a new drug could reduce the rate at which the disease develops by up to 75%, potentially allowing patients to maintain their independence and quality of life for longer periods.

The clinical trial involved 29 patients who received DNA injections directly into the brain, with significant health improvements observed after 36 months. The gene therapy company Unicure, which oversaw the study, plans to seek accelerated approval for the drug from US regulators.

UK scientists slow Huntington's disease for first time

A potentially ground-breaking advance in the fight against Huntington's disease. According to a recent study, a new drug could be able to reduce the speed at which the neurodegenerative condition develops by up to 75%. What the data means is that for a person who has Huntington's disease, they will be able to maintain their function longer, they'll be able to stay and work longer, they'll be able to support their families longer, they'll be able to be independent. A rare inherited brain disorder, Huntington's disease affects movement, thinking and mood and generally leads to death 10 to 30 years after the first symptoms appear. Until now, no official treatment has been approved, but the 29 patients who took part in the trial, receiving DNA injections directly into the brain, reported significant health improvements after 36 months. And so here is a picture of your brain, which I'm pleased to tell you looks fantastic. Huntington's patient Jack May-Davis, who lost his father and grandmother to the disease, says the treatment could help give him a new lease on life. It does make the future seem a little bit brighter. It's definitely going to help with knowing that I have got more time here than perhaps I first thought. Unicure, the gene therapy company that oversaw the study, says it plans to submit an application to U.S. regulators for accelerated market approval of the new drug by early next year.

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Breakdown