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.
When I was 13, my family went from having two seemingly normal kids to two CF patients in a manner of a few short months -- and boy, did it change everything. Because of my late diagnosis, I faced a unique set of challenges that helped make me the strong person I am today.
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.
There have been a few moments over the past four years that have provided us with perfect opportunities to give both of our kids a fuller understanding of the multifaceted nature of cystic fibrosis.
When our son was diagnosed with CF, the clinic director sat with me for hours answering my questions. Since that day, I have wanted to bottle up his advice and become a voice of encouragement for new CF parents.
Earlier this month, we learned that the new CF drug Orkambi was approved by the FDA. This was such an exciting, memorable and emotional day for everyone in the CF community. We heard from many who were thrilled about the potential for this drug to help people with CF and also heard from those who would not benefit from this breakthrough. Following are some reactions from those this impacts the most: people with CF and their loved ones.