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CFTR Modulator Therapy for Cystic Fibrosis.

作者信息

Grasemann Hartmut

机构信息

From the Division of Respiratory Medicine, Department of Pediatrics, the Hospital for Sick Children and University of Toronto, Toronto.

出版信息

N Engl J Med. 2017 Nov 23;377(21):2085-2088. doi: 10.1056/NEJMe1712335. Epub 2017 Nov 3.

DOI:10.1056/NEJMe1712335
PMID:29099349
Abstract
摘要

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CFTR Modulator Therapy for Cystic Fibrosis.用于囊性纤维化的CFTR调节剂疗法。
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Triple CFTR Modulator Therapy for Cystic Fibrosis.用于囊性纤维化的三联CFTR调节剂疗法
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Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment.囊性纤维化跨膜电导调节因子修饰药物:囊性纤维化治疗的未来。
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CFTR mutations and cystic fibrosis.囊性纤维化跨膜传导调节因子突变与囊性纤维化
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New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator.调节和纠正囊性纤维化跨膜传导调节因子的新治疗方法。
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F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review.用于治疗囊性纤维化的F508del-囊性纤维化跨膜传导调节因子校正剂:专利综述
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Ivacaftor: the first therapy acting on the primary cause of cystic fibrosis.依伐卡托:首个作用于囊性纤维化主要病因的疗法。
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A little CFTR can change a lot: slowing cystic fibrosis progression.少量的囊性纤维化跨膜传导调节因子就能产生很大影响:减缓囊性纤维化的病情发展。
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Entering the era of highly effective CFTR modulator therapy.进入高效CFTR调节剂治疗时代。
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Lumacaftor/ivacaftor, a novel agent for the treatment of cystic fibrosis patients who are homozygous for the F580del CFTR mutation.鲁马卡托/依伐卡托,一种用于治疗F580del囊性纤维化跨膜传导调节因子(CFTR)突变纯合子的囊性纤维化患者的新型药物。
Expert Rev Clin Pharmacol. 2017 Oct;10(10):1055-1072. doi: 10.1080/17512433.2017.1378094. Epub 2017 Sep 22.

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