The Cystic Fibrosis Foundation recently signed on to a letter with more than 70 organizations in opposition to the Right to Try Act, which passed in the House of Representatives earlier this month.
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The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.
Tezacaftor/ivacaftor (Symdeko™) is approved for individuals with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 26 specified mutations -- regardless of their other mutation.
Today, the U.S. Food and Drug Administration approved the use of ivacaftor (Kalydeco®) for children with cystic fibrosis ages 1 to 2 who have at least one mutation that is responsive to ivacaftor.
Celtaxsys Inc. reported promising results today from an early stage clinical trial of a potential anti-inflammatory drug for people with cystic fibrosis.
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.