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...

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Detalles Bibliográficos
Autores principales: Guigui, S., Wang, J., Cohen, R.I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Case Report
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

Internet

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

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