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
PTC Therapeutics has discontinued development of ataluren as a potential treatment for people with cystic fibrosis caused by a nonsense mutation. The Cystic Fibrosis Foundation is supporting efforts to pursue treatments for people with CF who have a nonsense, splicing or other rare mutations.
March 3, 2017
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3 min read