graphic by By Lbudd14 (Own work) [CC BY-SA 3.0 (http://creativecommons.org/licenses/by-sa/3.0)], via Wikimedia Commons
Living With CFResearchRespiratory DiseaseTreatments

Outcomes and Tolerances in Cystic Fibrosis Treatment Lumacaftor/Ivacaftor

Last month, the results of a study were published that looked at the progress and side effects of the cystic fibrosis treatment lumacaftor/ivacaftor in 116 people with CF during a year after starting the medication. 46 (39.7%) people reported side effects related to lumacaftor/ivacaftor, and of the side effects, 82.2% …

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Clinical TrialsResearchRespiratory DiseaseTreatments

Orkambi Study On Children Age 6-12 With DDF508: Cystic Fibrosis Treatment

Results are in from a 24-week study of Orkambi (lumacaftor/ivacaftor – a medication used in cystic fibrosis treatment) in 58 children with cystic fibrosis age 6-12 with two copies of the F508-delta mutation. Researchers were looking to examine the safety and efficacy of the CFTR potentiator on this age group and …

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Living With CFNews & EventsResearchTreatments

Treatment for Cystic Fibrosis: 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 in treatment for Cystic Fibrosis. …

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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 Cystic Fibrosis 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 (a medication used in Cystic Fibrosis treatment) From the Lancet Respiratory Journal, 20 January 2016 Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in patients …

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