Positive results from a study of ivacaftor (Kalydeco®) in children ages 1 to 2 were announced today.
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Two Phase 3 clinical trials of tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) showed positive results, Vertex Pharmaceuticals announced today.
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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.
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