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Living With CFNews & EventsResearchTreatments

Vertex Acquires Concert’s CFTR Potentiator CTP-656

Vertex Pharmaceuticals recently purchased the Phase 2 cystic fibrosis potentiator drug CTP-656 from Concert Pharmaceuticals. The deal is estimated to be worth $250 million dollars. CTP-656 is intended to be a longer-acting, once daily version of Vertex’s drug ivacaftor, which aims to restore CFTR function. The press release is here. …

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GeneticsLiving With CFRespiratory DiseaseTreatments

Does Ivacaftor Affect Lung Bacteria in Cystic Fibrosis?

Ivacaftor has been shown to increase CFTR activity and lung function in people with Cystic Fibrosis and the G551D mutation, but until a recent study, scientists were not sure if ivacaftor changed the level of bacteria in the lungs. The results of a deeper study on ivacaftor have been published, …

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Living With CFResearchRespiratory DiseaseTreatments

CFTR Function and Cigarette Smoke

Scientists are looking at how the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) potentiator, Ivacaftor, might be used in COPD, specifically chronic bronchitis. They found that cigarette smoking reduces CFTR activity in lung tissue, and that exposing the lung cells directly to a CFTR potentiator reverses this effect. While the vast …

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Clinical TrialsLiving With CFTreatments

Lumacaftor/Ivacaftor longer trial on Heterozygous F508del Cystic Fibrosis Patients

At the end of November, the results of a clinical trial of Lumacaftor/Ivacaftor, also known as Orkambi, in CF patients with one F508Del mutation were published in the Annals of the American Thoracic Society. A past trial for Orkambi in this type of CF patient (heterozygous F508del) lasted 28 days …

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GeneticsLiving With CFResearchTreatments

Trial results of Ivacaftor in children ages 2-5

Results are out from a two part study involving children ages 2-5 with at least one gating mutation trying Ivacaftor. From the Lancet Respiratory Journal, 20 January 2016 Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in patients aged 6 years or older with a …

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