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The use of ivacaftor in CFTR mutations resulting in residual functioning protein
INTRODUCTION: Ivacaftor, a cystic fibrosis transmembrane regulator (CFTR) potentiator is currently approved for use in individuals with class III gating mutations and the R117H mutation, a non-gating mutation with residual functioning CFTR. Nevertheless, ivacaftor may also be effective in individual...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Elsevier
2016
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5079351/ https://www.ncbi.nlm.nih.gov/pubmed/27812499 http://dx.doi.org/10.1016/j.rmcr.2016.10.012 |