Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation

BACKGROUND: A new approach in the treatment of cystic fibrosis involves improving the function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro. METHODS: We...

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Main Authors: Accurso, Frank J., Rowe, Steven M., Clancy, J.P., Boyle, Michael P., Dunitz, Jordan M., Durie, Peter R., Sagel, Scott D., Hornick, Douglas B., Konstan, Michael W., Donaldson, Scott H., Moss, Richard B., Pilewski, Joseph M., Rubenstein, Ronald C., Uluer, Ahmet Z., Aitken, Moira L., Freedman, Steven D., Rose, Lynn M., Mayer-Hamblett, Nicole, Dong, Qunming, Zha, Jiuhong, Stone, Anne J., Olson, Eric R., Ordoñez, Claudia L., Campbell, Preston W., Ashlock, Melissa A., Ramsey, Bonnie W.
格式: Artigo
語言:Inglês
出版: 2010
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3148255/
https://ncbi.nlm.nih.gov/pubmed/21083385
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa0909825
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