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Experimental Drug Is First To Help Kids With Premature-Aging Disease


Researchers have found the first drug to treat progeria, an extremely rare genetic disease that causes children to age so rapidly that many die in their teens.
The drug, called lonafarnib, is not a cure. But in a study published Monday of 28 children, it reversed changes in blood vessels that usually lead to heart attacks and strokes.
The treatment also helped kids with the disease put on weight and improved the structure of their bones. Children with progeria have small fragile bodies and can weigh just a third of a typical child their age.
“This is a fantastic first step,” says Leslie Gordon, medical director for the Progeria Research Foundation and the mother of a child with progeria. A second study is under way to see whether combining lonafarnib with two other drugs will work even better, says Gordon, who is also a physician on the faculty of Boston Children’s Hospital and Brown University.
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