The Bittersweet Downside of CFTR Modulators
Nov 19, 2018 09:00 am | Tré LaRosa
I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three years now. My lung function has remained stable during that time. CFTR modulators are medications that “fix” the defect that causes cystic fibrosis. “Fixing” the defect means improving CFTR function. A person without cystic fibrosis (CF) has at […]
The post The Bittersweet Downside of CFTR Modulators appeared first on Cystic Fibrosis News Today. |
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Orkambi Closer to Being Approved in Europe to Treat Children Ages 2-5 With Cystic Fibrosis
Nov 19, 2018 09:00 am | Alice Melão
The European Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Orkambi (lumacaftor/ivacaftor) as a treatment for children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals announced. CHMP’s positive opinion will be taken into consideration by the European Medicines Agency, which will now decide if the Orkambi label should be […]
The post Orkambi Closer to Being Approved in Europe to Treat Children Ages 2-5 With Cystic Fibrosis appeared first on Cystic Fibrosis News Today. |
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