PHASE 2 STUDIES REVEAL ADDITIVE EFFECTS OF VX-661, AN INVESTIGATIONAL CFTR CORRECTOR, AND IVACAFTOR, A CFTR POTENTIATOR, IN PATIENTS WITH CF WHO CARRY THE Delta F508-CFTR MUTATION

Pilewski Joseph M.; Donaldson Scott H.; Cooke Jon; Lekstrom-Himes Julie

首页> 外文期刊>Pediatric Pulmonology >PHASE 2 STUDIES REVEAL ADDITIVE EFFECTS OF VX-661, AN INVESTIGATIONAL CFTR CORRECTOR, AND IVACAFTOR, A CFTR POTENTIATOR, IN PATIENTS WITH CF WHO CARRY THE Delta F508-CFTR MUTATION

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PHASE 2 STUDIES REVEAL ADDITIVE EFFECTS OF VX-661, AN INVESTIGATIONAL CFTR CORRECTOR, AND IVACAFTOR, A CFTR POTENTIATOR, IN PATIENTS WITH CF WHO CARRY THE Delta F508-CFTR MUTATION

机译：第2阶段研究了携带Delta F508-CFTR突变的CF患者的研究性CFTR矫正器VX-661和CFTR矫正剂IVACAFTOR的揭示性增效作用

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《Pediatric Pulmonology》 |2014年第38期|共3页
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Pilewski Joseph M.; Donaldson Scott H.; Cooke Jon; Lekstrom-Himes Julie;
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1. PHASE 2 STUDIES REVEAL ADDITIVE EFFECTS OF VX-661, AN INVESTIGATIONAL CFTR CORRECTOR, AND IVACAFTOR, A CFTR POTENTIATOR, IN PATIENTS WITH CF WHO CARRY THE Delta F508-CFTR MUTATION [J] . Pilewski Joseph M., Donaldson Scott H., Cooke Jon, Pediatric Pulmonology . 2014,第Suppla38期

机译：第2阶段研究了携带Delta F508-CFTR突变的CF患者的研究性CFTR矫正器VX-661和CFTR矫正剂IVACAFTOR的揭示性增效作用
2. 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 [J] . BoyleM.P., BellS.C., KonstanM.W., The lancet. Respiratory medicine. . 2014,第7期

机译：CFTR校正剂（lumacaftor）和CFTR增强剂（ivacaftor）用于治疗phe508del CFTR突变的囊性纤维化患者：2期随机对照试验
3. EFFECT OF LUMACAFTOR IN COMBINATION WITH IVACAFTOR IN PATIENTS WITH CF WHO ARE HOMOZYGOUS FOR Delta F508-CFTR: PHASE 3 TRAFFIC AND TRANSPORT STUDIES [J] . Wainwright Claire E., Elborn J. Stuart, Ramsey Bonnie, Pediatric Pulmonology . 2014,第Suppla38期

机译：氟马考特联合依伐他汀对CF F相同的CF患者的影响：第三阶段交通和运输研究
4. Functional studies of rare CFTR missense mutations facilitate interpretation of genotype-phenotype relationships. [D] . Krasnov, Kristina V. 2009

机译：罕见CFTR错义突变的功能研究有助于解释基因型与表型的关系。
5. Therapeutic benefit observed with the CFTR potentiator ivacaftor in a CF patient homozygous for the W1282X CFTR nonsense mutation [O] . Venkateshwar Mutyam, Emily Falk Libby, Ning Peng, -1

机译：CFTR增强剂ivacaftor对W1282X CFTR无意义突变纯合CF患者的治疗效果
6. WS7.3 VX-661, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the F508Del-CFTR mutation: Interim analysis [O] . Donaldson S., Pilewski J., Griese M., 2013

机译：WS7.3 VX-661，研究性CFTR校正剂，与ivacaftor（CFTR增强剂）组合，用于CF和F508Del-CFTR突变纯合子患者：中期分析

PHASE 2 STUDIES REVEAL ADDITIVE EFFECTS OF VX-661, AN INVESTIGATIONAL CFTR CORRECTOR, AND IVACAFTOR, A CFTR POTENTIATOR, IN PATIENTS WITH CF WHO CARRY THE Delta F508-CFTR MUTATION

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