WS7.3 VX-661, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the F508Del-CFTR mutation: Interim analysis
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This article is published in Journal of Cystic Fibrosis.The article was published on 2013-06-01 and is currently open access. It has received 33 citations till now. The article focuses on the topics: Ivacaftor.read more
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Journal ArticleDOI
New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls
TL;DR: Combination drug treatment with correctors and potentiators appears to be required to restore CFTR function of F508del, the most common CFTR mutation.
Journal ArticleDOI
Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation
Daniela De Stefano,Valeria Rachela Villella,Speranza Esposito,Antonella Tosco,Angela Sepe,Fabiola De Gregorio,Laura Salvadori,R. Grassia,Carlo A Leone,Giuseppe De Rosa,Maria Chiara Maiuri,Massimo Pettoello-Mantovani,Stefano Guido,Anna Bossi,A. Zolin,Andrea Venerando,Lorenzo A. Pinna,Anil Mehta,Gianni Bona,Guido Kroemer,Luigi Maiuri,Valeria Raia +21 more
TL;DR: It is reported that oral treatment with cysteamine greatly reduces the mortality rate and improves the phenotype of newborn mice bearing the F508del-CFTR mutation, and optimal schedules of cysteamines plus EGCG might be used for the treatment of CF caused by the F504del- CFTR mutation.
Journal ArticleDOI
Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients.
TL;DR: Functional assessment of patients' tissues ex vivo is key for diagnosing and predicting the severity of this disease, and will also be crucial to assess drug responses, in order to effectively treat all CF patients.
Journal ArticleDOI
State of progress in treating cystic fibrosis respiratory disease.
TL;DR: The current knowledge of the pathophysiology of lung disease in patients with CF is described, along with the current treatment of CF lung disease, and the therapies in development that offer great promise to patients are described.
Journal Article
CFTR Modulators for the Treatment of Cystic Fibrosis.
Rebecca S. Pettit,Chris Fellner +1 more
TL;DR: Although ivacaftor is currently the only FDA-approved CFTR modifier, others are in development, and the market for other CFTR modifiers is expected to grow.
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