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Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del
Author(s) -
Jennifer L. TaylorCousar,
À. Munck,
Edward F. McKone,
Cornelis K. van der Ent,
Alexander Mœller,
C Simard,
Linda T. Wang,
Edward P. Ingenito,
Charlotte McKee,
Yimeng Lu,
Julie Lekstrom-Himes,
J.S. Elborn
Publication year - 2017
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/nejmoa1709846
Subject(s) - ivacaftor , medicine , cystic fibrosis , placebo , clinical endpoint , randomization , cystic fibrosis transmembrane conductance regulator , randomized controlled trial , pathology , alternative medicine
Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis.

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