lunes, 19 de noviembre de 2018

Cystic Fibrosis News Today Weekly Digest


Cystic Fibrosis Weekly Update

Contents:

The Bittersweet Downside of CFTR Modulators

Nov 19, 2018 09:00 am | Tré LaRosa



modulatorsI’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 […]
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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



Orkambi CHMP EuropeThe 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 […]
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