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Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study.

BACKGROUND: Ivacaftor is generally safe and effective in patients 2 years and older with cystic fibrosis (CF) and specific CFTR mutations. We evaluated the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of ivacaftor in children aged 12 to <24 months. METHODS: This...

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書目詳細資料
發表在:Lancet Respir Med
Main Authors: Rosenfeld, Margaret, Wainwright, Claire E., Higgins, Mark, Wang, Linda T., McKee, Charlotte, Campbell, Daniel, Tian, Simon, Schneider, Jennifer, Cunningham, Steve, Davies, Jane C.
格式: Artigo
語言:Inglês
出版: 2018
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6626762/
https://ncbi.nlm.nih.gov/pubmed/29886024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S2213-2600(18)30202-9
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