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Early findings from a clinical trial suggest that a new gene therapy, administered directly into the brain, may slow the progression of Huntington's disease. The therapy targets the faulty gene responsible for the condition, aiming to reduce the production of the toxic protein that damages brain cells. While the trial involved only a small number of participants, the results represent an important step toward developing the first treatment to alter the course of the disease, which currently has no cure and is typically managed only through supportive care.
Source: https://www.bbc.com/news/articles/cevz13xkxpro
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Discussion forums: Research: BBC News: Huntington's disease successfully treated for first time
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