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CFTR


On July 2, 2015 the FDA announced its approval for the potent duo of Lumacaftor and Ivacaftor to treat patients suffering from Cystic Fibrosis (CF). Coined Orkambi, which is marketed by Vertex Pharmaceuticals, this effectual treatment is specifically approved for patients ages 12 and older who have the homozygous F508 mutation of the CF transmembrane[…]

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Known as the brand name Cystic Fibrosis drug Kalydeco, marketed by Vertex Pharmaceuticals, Ivacaftor recently received an MAA line extension. As of June 2015 children ages two through five who have specific mutations in the CFTR gene may be administered Ivacaftor as therapy. As a unique, tolerable and effectual treatment Ivacaftor acts as a novel[…]

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Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic Fibrosis. Ataluren also appears to be particularly effective for patients with Cystic Fibrosis (CF) nonsense mutation. This genetic mutation interrupts the CFTR protein[…]

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