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Experimental Gene Therapy Successfully Treats Huntington's Disease for the First Time

A clinical trial at University College London showed gene therapy slowed Huntington’s disease progression by 75% in 29 patients, offering a potential one-time treatment.

Summary by Smithsonian Mag
Preliminary results from a small study offer hope for treatment of the degenerative disease

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Chorea Huntington unstoppably disintegrates the brain. Now researchers have succeeded in slowing down the course of the dreaded disease.

·Germany
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It is a gene therapy, the first to be tested with these patients, which is given with brain surgery that lasts between 12 and 18 hours. More information: Beyond the ALS: the other rare (and devastating) diseases that 'nobody' talks about in Spain

·Spain
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British researchers have managed for the first time to slow down the progression of Huntington's disease in a handful of patients, which is a rare glimmer of hope for those with this debilitating neurodegenerative disease.

A new gene therapy could extend the lives of patients with Huntington's disease by years or even decades, according to a study by British researchers.

·Hungary
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Nature broke the news in United Kingdom on Thursday, September 25, 2025.
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