Abstract
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the “magic-bullet” cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
| Original language | English |
|---|---|
| Pages (from-to) | 130-141 |
| Number of pages | 12 |
| Journal | Clinical Pharmacology and Therapeutics |
| Volume | 101 |
| Issue number | 1 |
| DOIs | |
| Publication status | Published - 1 Jan 2017 |
Cite this
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Can cystic fibrosis patients finally catch a breath with lumacaftor/ivacaftor? / Schneider, E K; Reyes-Ortega, Felisa; Li, Jian; Velkov, Tony.
In: Clinical Pharmacology and Therapeutics, Vol. 101, No. 1, 01.01.2017, p. 130-141.Research output: Contribution to journal › Review Article › Research › peer-review
TY - JOUR
T1 - Can cystic fibrosis patients finally catch a breath with lumacaftor/ivacaftor?
AU - Schneider, E K
AU - Reyes-Ortega, Felisa
AU - Li, Jian
AU - Velkov, Tony
PY - 2017/1/1
Y1 - 2017/1/1
N2 - Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the “magic-bullet” cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
AB - Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the “magic-bullet” cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
UR - http://www.scopus.com/inward/record.url?scp=85006036356&partnerID=8YFLogxK
U2 - 10.1002/cpt.548
DO - 10.1002/cpt.548
M3 - Review Article
VL - 101
SP - 130
EP - 141
JO - Clinical Pharmacology and Therapeutics
JF - Clinical Pharmacology and Therapeutics
SN - 0009-9236
IS - 1
ER -