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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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| 發表在: | Lancet Respir Med |
|---|---|
| Main Authors: | , , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
2018
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| 主題: | |
| 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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