Chicago (AP) – Children with a rare, incurable disease that causes rapid aging and early death may live longer if treated with an experimental drug first developed for cancer patients.
That’s according to small, preliminary study in 27 kids with progeria. The benefit was small: Treated children were more likely to survive during the two-year study than children who didn’t get the drug.
But some kids taking the drug lonafarnib (lohn-uh-FAR-nib) in this and other studies have lived into their late teens. The researchers say the results suggest a potential breakthrough for a heartbreaking condition.
The drug helps disable a protein that accumulates abnormally in progeria and causes cell damage. The genetic disease affects about 1 in 20 million people.
The study was published Tuesday in the Journal of the American Medical Association.
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