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Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR
Rationale: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. Objectives: To evaluate the safety and...
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| 發表在: | Am J Respir Crit Care Med |
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| Main Authors: | , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Thoracic Society
2018
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5768901/ https://ncbi.nlm.nih.gov/pubmed/28930490 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1164/rccm.201704-0717OC |
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