Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel. F508del-CFTR, the most common CF-causing mutation (found in ∼90% of patients), causes severe processing and trafficking...

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Auteurs principaux:	Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe
Format:	Artigo
Langue:	Inglês
Publié:	European Respiratory Society 2019-06-01
Collection:	ERJ Open Research
Accès en ligne:	http://openres.ersjournals.com/content/5/2/00082-2019.full
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