New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
July 18, 2017
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6 min read
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
May 17, 2017
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3 min read