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Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation
BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population. METHODS: We conducted a multicenter, prospe...
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| Udgivet i: | J Cyst Fibros |
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| Main Authors: | , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2020
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8183611/ https://ncbi.nlm.nih.gov/pubmed/33249004 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jcf.2020.11.008 |
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