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Experimental Gene Therapy Found To Slow Huntington's Disease Progression


Doctors report the first successful treatment for Huntington's disease using a new type of gene therapy given during 12 to 18 hours of delicate brain surgery. The BBC reports: An emotional research team became tearful as they described how data shows the disease was slowed by 75% in patients. It me...

Doctors report the first successful treatment for Huntington's disease using a new type of gene therapy given during 12 to 18 hours of delicate brain surgery. The data showed that three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life. Levels of neurofilaments in spinal fluid -- a clear sign of brain cells dying -- should have increased by a third if the disease continued to progress, but was actually lower than at the start of the trial.

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