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Expert Review of Respiratory Medicine Volume 10, 2016 - Issue 1
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Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction

John J. BrewingtonDivision of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center and the University of Cincinnati, Cincinnati, OH, USA
,
Gary L. McPhailDivision of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center and the University of Cincinnati, Cincinnati, OH, USA
&
John P. ClancyDivision of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center and the University of Cincinnati, Cincinnati, OH, USACorrespondence[email protected]
Pages 5-17 | Published online: 09 Dec 2015

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Sarah L. Paterson, Peter J. Barry & Alexander R. Horsley. (2020) Tezacaftor and ivacaftor for the treatment of cystic fibrosis. Expert Review of Respiratory Medicine 14:1, pages 15-30.
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Jane C. Davies & Isaac Martin. (2018) New anti-pseudomonal agents for cystic fibrosis- still needed in the era of small molecule CFTR modulators?. Expert Opinion on Pharmacotherapy 19:12, pages 1327-1336.
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Sara M. Zak, John P. Clancy & John J. Brewington. (2017) CFTR functional assays in drug development. Expert Opinion on Orphan Drugs 5:11, pages 889-898.
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Kristin M. Hudock & John Paul Clancy. (2017) An update on new and emerging therapies for cystic fibrosis. Expert Opinion on Emerging Drugs 22:4, pages 331-346.
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Deborah M. Cholon, Charles R. Esther$suffix/text()$suffix/text() & Martina Gentzsch. (2016) Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation. Expert Review of Precision Medicine and Drug Development 1:3, pages 235-243.
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Articles from other publishers (12)

Insa Korten, Elisabeth Kieninger, Linn Krueger, Marina Bullo, Christa E. Flück, Philipp Latzin, Carmen Casaulta & Claudia Boettcher. (2022) Short-Term Effects of Elexacaftor/Tezacaftor/Ivacaftor Combination on Glucose Tolerance in Young People With Cystic Fibrosis—An Observational Pilot Study. Frontiers in Pediatrics 10.
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Lumír Kunovský, Petr Dítě, Petr Jabandžiev, Michal Eid, Karolina Poredská, Jitka Vaculová, Dana Sochorová, Pavel Janeček, Pavla Tesaříková, Martin Blaho, Jan Trna, Jan Hlavsa & Zdeněk Kala. (2021) Causes of Exocrine Pancreatic Insufficiency Other Than Chronic Pancreatitis. Journal of Clinical Medicine 10:24, pages 5779.
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Saangyoung E. Lee, Zainab Farzal, M.Leigh Anne Daniels, Brian D. Thorp, Adam M. Zanation, Brent A. Senior, Charles S. EbertJrJr & Adam J. Kimple. (2020) Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy: A Review for the Otolaryngologist. American Journal of Rhinology & Allergy 34:4, pages 573-580.
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Masood Khaksar Toroghi, William R. Cluett & Radhakrishnan Mahadevan. (2020) A Personalized Multiscale Modeling Framework for Dose Selection in Precision Medicine. Industrial & Engineering Chemistry Research 59:21, pages 9819-9829.
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Onofrio Laselva, Tracy A. Stone, Christine E. Bear & Charles M. Deber. (2020) Anti-Infectives Restore ORKAMBI® Rescue of F508del-CFTR Function in Human Bronchial Epithelial Cells Infected with Clinical Strains of P. aeruginosa. Biomolecules 10:2, pages 334.
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Angel Li, Tim Vigers, Laura Pyle, Edith Zemanick, Kristen Nadeau, Scott D. Sagel & Christine L. Chan. (2019) Continuous glucose monitoring in youth with cystic fibrosis treated with lumacaftor-ivacaftor. Journal of Cystic Fibrosis 18:1, pages 144-149.
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Damien Adam, Claudia Bilodeau, Laura Sognigbé, Émilie Maillé, Manon Ruffin & Emmanuelle Brochiero. (2018) CFTR rescue with VX-809 and VX-770 favors the repair of primary airway epithelial cell cultures from patients with class II mutations in the presence of Pseudomonas aeruginosa exoproducts. Journal of Cystic Fibrosis 17:6, pages 705-714.
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John J. Brewington, Jessica Backstrom, Amanda Feldman, Elizabeth L. Kramer, Jessica D. Moncivaiz, Alicia J. Ostmann, Xiaoting Zhu, L. Jason Lu & John P. Clancy. (2018) Chronic β2AR stimulation limits CFTR activation in human airway epithelia. JCI Insight 3:4.
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Bright I. Nwaru, Charles Friedman, John Halamka & Aziz Sheikh. (2017) Can learning health systems help organisations deliver personalised care?. BMC Medicine 15:1.
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Alison E. Fohner, Ellen M. McDonagh, John P. Clancy, Michelle Whirl Carrillo, Russ B. Altman & Teri E. Klein. (2017) PharmGKB summary. Pharmacogenetics and Genomics 27:1, pages 39-42.
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EK Schneider, F Reyes-Ortega, J Li & T Velkov. (2017) Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?. Clinical Pharmacology & Therapeutics 101:1, pages 130-141.
Crossref
Euan A. Ashley. (2016) Towards precision medicine. Nature Reviews Genetics 17:9, pages 507-522.
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