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VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
Author(s) -
Jane C. Davies,
Samuel M. Moskowitz,
Cynthia D. Brown,
Alex Horsley,
Marcus Mall,
Edward F. McKone,
Barry J. Plant,
Dario Prais,
Bonnie W. Ramsey,
Jennifer L. TaylorCousar,
Elizabeth Tullis,
Ahmet Uluer,
Charlotte McKee,
Sarah Robertson,
Rebecca A. Shilling,
C Simard,
Fredrick Van Goor,
David Waltz,
Fengjuan Xuan,
Timothy Young,
Steven M. Rowe
Publication year - 2018
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/nejmoa1807119
Subject(s) - ivacaftor , allele , cystic fibrosis , medicine , genetics , biology , gene , cystic fibrosis transmembrane conductance regulator
The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659-tezacaftor-ivacaftor), was developed to restore the function of Phe508del CFTR protein in patients with cystic fibrosis.

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