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.
Celtaxsys Inc. reported promising results today from an early stage clinical trial of a potential anti-inflammatory drug for people with cystic fibrosis.
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.
As a teacher with cystic fibrosis, I find it no surprise that heading back to school can be a shock to my system. But over the years, I've learned that if I can remember three main things, I can stay healthy through the transition back to school.
During my five months in Denmark for a study abroad program, I not only learned more about myself and others, but I became more confident in my ability to take care of myself. Don't let CF stand in the way of experiencing that, or any other dreams.
Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.