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FDA Approves First Targeted Drug against Cystic Fibrosis

Image: Illustration by John Hendrix The U.S. Food and Drug Administration today approved a new drug that tackles the underlying cause of cystic fibrosis (CF) in 4 percent of patients . The drug, called ivacaftor (brand name Kalydeco), acts by helping the body make better use of a protein that works incorrectly in cystic fibrosis patients.

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Video: Breakthrough in cystic fibrosis treatment

The new drug Kalydeco is being hailed as a breakthrough in the treatment of cystic fibrosis, targeting the underlying genetic cause of the disease decreasing the symptoms. Dr. Jon LaPook reports.

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