Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508delCFTR | Zendy

Claire Wainwright | Zendy; J.S. Elborn | Zendy; Bonnie W. Ramsey | Zendy; Gautham Marigowda | Zendy; Xiaohong Huang | Zendy; Marco Cipolli | Zendy; Carla Colombo | Zendy; Jane C. Davies | Zendy; K. De Boeck | Zendy; Patrick A. Flume | Zendy; Michael W. Konstan | Zendy; Susanna A. McColley | Zendy; Karen McCoy | Zendy; Edward F. McKone | Zendy; À. Munck | Zendy; Félix Ratjen | Zendy; Steven M. Rowe | Zendy; David Waltz | Zendy; Michael Boyle | Zendy

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Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508delCFTR

Author(s) -

Claire Wainwright,

J.S. Elborn,

Bonnie W. Ramsey,

Gautham Marigowda,

Xiaohong Huang,

Marco Cipolli,

Carla Colombo,

Jane C. Davies,

K. De Boeck,

Patrick A. Flume,

Michael W. Konstan,

Susanna A. McColley,

Karen McCoy,

Edward F. McKone,

À. Munck,

Félix Ratjen,

Steven M. Rowe,

David Waltz,

Michael Boyle

Publication year - 2015

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1409547

Subject(s) - ivacaftor , medicine , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , placebo , randomization , clinical endpoint , gastroenterology , randomized controlled trial , urology , surgery , pathology , alternative medicine

Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR mutation.

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