Gene therapy slows Huntington's disease progression in trials

bbc.com

Huntington's disease has been successfully treated for the first time, with a new gene therapy significantly slowing its progression. The experimental treatment, delivered via brain surgery, reduced disease progression by an average of 75% in trial patients, potentially extending quality of life for decades. This breakthrough offers hope for the thousands affected by the devastating inherited neurological disorder, with regulatory applications expected in the US in 2026.


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Gene therapy slows Huntington's disease progression in trials | News Minimalist