Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR

Weiqiang Zhang, X. Zhang, Yanhui Zhang, D. C. Stokes, A. P. Naren

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

Cystic fibrosis (CF) is a life-shortening inherited disease caused by the loss or dysfunction of the CF transmembrane conductance regulator (CFTR) channel activity resulting from mutations in the CFTR gene. Phe508del is the most prevalent mutation, with approximately 90% of all CF patients carrying it on at least one allele. Over the past two or three decades, significant progress has been made in understanding the pathogenesis of CF, and in the development of effective CF therapies. The approval of Orkambi® (lumacaftor/ivacaftor) marks another milestone in CF therapeutics development, which, with the advent of personalized medicine, could potentially revolutionize CF care and management. This article reviews the rationale, progress and future direction in the development of lumacaftor/ivacaftor combination to treat CF patients homozygous for the Phe508del-CFTR mutation.

Original languageEnglish (US)
Pages (from-to)229-237
Number of pages9
JournalDrugs of Today
Volume52
Issue number4
DOIs
StatePublished - Apr 1 2016

Fingerprint

Cystic Fibrosis
Mutation
Cystic Fibrosis Transmembrane Conductance Regulator
Precision Medicine
Regulator Genes
ivacaftor drug combination lumacaftor
Alleles
Therapeutics

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Pharmacology (medical)

Cite this

Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR. / Zhang, Weiqiang; Zhang, X.; Zhang, Yanhui; Stokes, D. C.; Naren, A. P.

In: Drugs of Today, Vol. 52, No. 4, 01.04.2016, p. 229-237.

Research output: Contribution to journalArticle

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