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.
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.
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