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Ivacaftor in a young boy with the rare gating mutation S549R - use of lung clearance index to track progress: a case report
BACKGROUND: Ivacaftor acts as a potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) and increases the transepithelial chloride transport of CFTR in 9 of 10 known gating mutations causing cystic fibrosis. S549R is a rare gating mutation considered to be less sensitive to pot...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4609097/ https://www.ncbi.nlm.nih.gov/pubmed/26474553 http://dx.doi.org/10.1186/s12890-015-0120-1 |