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Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.

作者信息

Wainwright Claire E, Elborn J Stuart, Ramsey Bonnie W

出版信息

N Engl J Med. 2015 Oct 29;373(18):1783-4. doi: 10.1056/NEJMc1510466.

DOI:10.1056/NEJMc1510466
PMID:26510034
Abstract
摘要

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Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.针对携带Phe508del CFTR纯合突变的囊性纤维化患者使用鲁马卡托-依伐卡托。
N Engl J Med. 2015 Oct 29;373(18):1783-4. doi: 10.1056/NEJMc1510466.
2
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.鲁马卡托-依伐卡托用于携带苯丙氨酸508缺失（Phe508del）囊性纤维化跨膜传导调节因子（CFTR）纯合突变的囊性纤维化患者
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3
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.鲁马卡托-依伐卡托用于携带苯丙氨酸508位缺失CFTR基因纯合突变的囊性纤维化患者。
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A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.一种用于治疗携带 phe508del CFTR 突变的囊性纤维化患者的 CFTR 校正剂（芦卡帕替尼）和 CFTR 增强剂（依伐卡托）：一项 2 期随机对照试验。
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Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR.鲁马卡托/依伐卡托联合用药治疗携带Phe508del-CFTR纯合突变的囊性纤维化患者。
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Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis.依库珠单抗/ivacaftor 联合治疗对肺功能亚组纯合子 Phe508del CFTR 囊性纤维化患者的疗效和安全性：一项汇总分析。
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Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial.在 F508del-CFTR 纯合子的 6-11 岁囊性纤维化患者中， lumacaftor 和 ivacaftor 的疗效和安全性：一项随机、安慰剂对照的 3 期临床试验。
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Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation.预测鲁马卡托/依伐卡托对纯合子Phe508del突变囊性纤维化患者的长期临床和经济结局
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