A group of doctors in London say Huntington’s disease (HD) has been successfully treated for the first time, offering new hope against the fatal genetic disorder. A gene therapy trial at University College London showed the disease’s progression slowed by 75% in 29 patients, according to results released on September 23.
The treatment, delivered in a single 12- to 18-hour brain surgery, uses a modified virus to silence the faulty huntingtin (HTT) gene that kills brain cells. Researchers said the results could mean decades of added quality of life and, if administered early, may even prevent symptoms.
HD is a rare, inherited disorder that causes the progressive breakdown of nerve cells in the brain. It's a condition that's passed down in families from generation to generation, with symptoms first occurring in people in their thirties and forties, though children are also known to develop the disease. There is currently no cure for Huntington's, and it's usually fatal after a period of up to 20 years—but this new breakthrough could transform outcomes for thousands of families worldwide.
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