I am both excited and scared that my son will be old enough to try Trikafta when he turns 12 later this year. In some ways, I can hardly wait to see how he will benefit. But I also know that he might experience side effects, and I don't want him to have any setbacks.
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Does Trikafta® increase the chance of pregnancy for women with cystic fibrosis? Can men regain their fertility on modulators? I recently had a candid chat with the chair of the Women's Health Research Working Group to find out the answers to these and other questions about reproductive health.
As I listened to the second plenary for this year's North American Cystic Fibrosis Conference, I couldn't help but think of my own experience with CFTR modulators and the positive effects that they have had on my health.
Having been diagnosed with cystic fibrosis as a baby, I grew up thinking I had an early expiration date. Once I started taking Kalydeco®, my health completely changed for the better. For the first time, I had a solid future ahead of me, and I needed to prepare for it.
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
I had to stop taking Orkambi® because my body couldn't handle it. I'm having a different -- better -- experience with Symdeko™.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
“For the first time in over a decade, a striking silence fills the Cheevers' barn-style home,” begins a story in the September issue of the science magazine Discover.
The Institute for Clinical and Economic Review (ICER) recently posted a draft evidence report to outline their initial assessment of the clinical effectiveness and value of CFTR modulators, including elexacaftor/tezacaftor/ivacaftor (TrikaftaTM). The document is open for public comment through March 18.
This morning, Vertex Pharmaceuticals announced the results from the Phase 3 clinical trials of ivacaftor (Kalydeco™) and lumacaftor (VX-809) in people with two copies of the F508del mutation.