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.
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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.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, announced today a $15 million research agreement with biopharmaceutical company Shire plc to support the development of a new cystic fibrosis treatment targeting the underlying cause of the disease.