-
Clin. Pharmacol. Ther. · Jan 2017
ReviewCan Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
- E K Schneider, F Reyes-Ortega, J Li, and T Velkov.
- Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences; Monash University, Parkville, Victoria, Australia.
- Clin. Pharmacol. Ther. 2017 Jan 1; 101 (1): 130-141.
AbstractCystic 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.© 2016 American Society for Clinical Pharmacology and Therapeutics.
Notes
Knowledge, pearl, summary or comment to share?You can also include formatting, links, images and footnotes in your notes
- Simple formatting can be added to notes, such as
*italics*,_underline_or**bold**. - Superscript can be denoted by
<sup>text</sup>and subscript<sub>text</sub>. - Numbered or bulleted lists can be created using either numbered lines
1. 2. 3., hyphens-or asterisks*. - Links can be included with:
[my link to pubmed](http://pubmed.com) - Images can be included with:
 - For footnotes use
[^1](This is a footnote.)inline. - Or use an inline reference
[^1]to refer to a longer footnote elseweher in the document[^1]: This is a long footnote..