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.
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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.
Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies
Finding a Balance Between CF and High School
Growing up, I never had any doubts that I would one day go off to college and pursue a career, despite having cystic fibrosis. But when the time came to prepare to attend Marquette University and live on my own in Milwaukee, I knew I would need a plan in order to make my transition to college life as smooth as possible.