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Safety and efficacy of treatment with lumacaftor in combination with ivacaftor in younger patients with cystic fibrosis

INTRODUCTION: Cystic fibrosis (CF) is the most common autosomal recessive disorder affecting approximately 70,000 people worldwide. The lack of functional cystic fibrosis transmembrane conductance regulator (CFTR) causes dysregulation of epithelial fluid transport in the lungs, gastrointestinal trac...

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Publicado en:Expert Rev Respir Med
Autores principales: Cheng, Pi Chun, Alexiou, Stamatia, Rubenstein, Ronald C.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2019
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6482058/
https://ncbi.nlm.nih.gov/pubmed/30929526
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/17476348.2019.1602040
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