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CFTR Modulator Therapies in Pediatric Cystic Fibrosis: Focus on Ivacaftor
INTRODUCTION: Mutations in the cystic fibrosis transmembrane conductance regulator protein (CFTR) cause cystic fibrosis (CF), a disease with life threatening pulmonary and gastrointestinal manifestations. Recent breakthrough therapies restore function to select disease-causing CFTR mutations. Ivacaf...
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| Udgivet i: | Expert Opin Orphan Drugs |
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| Main Authors: | , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2016
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5193376/ https://ncbi.nlm.nih.gov/pubmed/28042521 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2016.1226800 |
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