The Institute for Clinical and Economic Review (ICER) developed an assessment of the comparative clinical effectiveness and value of cystic fibrosis therapies. The Foundation provided input to help inform this process.
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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.
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 Cystic Fibrosis Foundation's Greater New York Chapter recently recognized leading journalist Scott Pelley at the 14th annual Breath of Life Gala for his unwavering dedication to those living with CF.
The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of lumacaftor/ivacaftor (Orkambi®) and ivacaftor/tezacaftor (VX-661). The Foundation is sharing input throughout the process to help incorporate the patient and clinical perspective.
The U.S. House of Representatives has passed the American Health Care Act (AHCA), legislation that fails to adequately protect people living with cystic fibrosis.
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.