Written by
The Associated Press
WASHINGTON (AP) -- The first drug that treats the root cause of cystic fibrosis won approval Tuesday, offering a life-changing treatment for a handful of patients with the deadly illness, and broader hope for thousands more patients suffering from the inherited disease.
The Food and Drug approved Vertex Pharmaceutical's Kalydeco (kuh-LYE-deh-koh) to improve lung function in patients with a rare variant of the disease that affects just 1,200 people in the U.S. or about 4 percent of the affected population nationwide. These patients have a protein defect that prevents their cells from properly absorbing and excreting salt and water.
About 30,000 Americans live with cystic fibrosis, a disease that causes sticky mucus buildup in the lungs and other organs, leading to infections, digestive problems and death in young adulthood.
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