Groundbreaking Gene Therapy Slows Huntington’s Disease for the First Time

source: nicenews.com  |  image:  pexels.com

It doesn’t get more “nice news” than this: In a historic medical first, a pioneering gene therapy successfully slowed the progression of Huntington’s disease by 75% in a clinical trial. The breakthrough offers new hope for the tens of thousands living with the devastating hereditary condition, which progressively destroys brain cells and impairs movement, cognition, and behavior — and until now, there was no treatment capable of slowing or halting its advance.
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“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” Sarah Tabrizi, the trial co-lead, told the BBC. The study involved 29 patients who underwent a complex 12- to 18-hour surgery to deliver a virus with a specially designed sequence of DNA directly into the brain, targeting (and reducing) the mutated “huntingtin” protein that drives the progression of the disease.
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​Patients receiving the highest doses of the therapy showed significant improvement on tests of motor and cognitive function, and reported improved day-to-day life experiences — a profound outcome. Fellow lead researcher Ed Wild explained: “There was every chance that we would never see a result like this, so to be living in a world where we know this is not only possible, but the actual magnitude of the effect is breathtaking; it’s very difficult to fully encapsulate the emotion.”
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Currently, the therapy is very costly, but the company developing it plans to seek FDA approval in the U.S. in early 2026 to make it more widely available. Learn more about the life-changing treatment.​