Cystic Fibrosis Drug Shows Rapid Benefits
Wednesday, November 2, 2011 - 16:31
in Health & Medicine
People with an uncommon form of cystic fibrosis started gaining weight and were better able to breathe than their untreated counterparts after just two weeks on an experimental drug, according to a study published November 2 in the New England Journal of Medicine . If approved by the U.S. Food and Drug Administration, the drug, once known as VX-770 and now called ivacaftor, would be the first medication to target the underlying protein defect that causes the disease.The findings are particularly compelling because the drug's benefits lasted for the duration of the 48-week randomized double-blind, placebo-controlled trial (the most rigorous type of scientific investigation that can be conducted). [More]