A new phase of CFTR treatment for cystic fibrosis?

被引：1

作者：

Bell, Scott C. ^{[1
,2
]}

机构：

[1] QIMR Berghofer Med Res Inst, Brisbane, Qld 4006, Australia

[2] Prince Charles Hosp, Brisbane, Qld 4032, Australia

来源：

LANCET RESPIRATORY MEDICINE | 2015年 / 3卷 / 09期

关键词：

CLINICAL-TRIALS NETWORK; G551D MUTATION; IVACAFTOR; GENE; EFFICACY; SAFETY;

D O I：

10.1016/S2213-2600(15)00282-9

中图分类号：

R4 [临床医学];

学科分类号：

1002 ; 100602 ;

摘要：

引用

页码：662 / 663

页数：2

共 15 条

[11] Improving rate of decline of FEV1 in young adults with cystic fibrosis [J].

Que, C ;

Cullinan, P ;

Geddes, D .

THORAX, 2006, 61 (02) :155-157

[12] A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation [J].

Ramsey, Bonnie W. ;

Davies, Jane ;

McElvaney, N. Gerard ;

Tullis, Elizabeth ;

Bell, Scott C. ;

Drevinek, Pavel ;

Griese, Matthias ;

McKone, Edward F. ;

Wainwright, Claire E. ;

Konstan, Michael W. ;

Moss, Richard ;

Ratjen, Felix ;

Sermet-Gaudelus, Isabelle ;

Rowe, Steven M. ;

Dong, Qunming ;

Rodriguez, Sally ;

Yen, Karl ;

Ordonez, Claudia ;

Elborn, J. Stuart .

NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (18) :1663-1672

[13] IDENTIFICATION OF THE CYSTIC-FIBROSIS GENE - CHROMOSOME WALKING AND JUMPING [J].

ROMMENS, JM ;

IANNUZZI, MC ;

KEREM, BS ;

DRUMM, ML ;

MELMER, G ;

DEAN, M ;

ROZMAHEL, R ;

COLE, JL ;

KENNEDY, D ;

HIDAKA, N ;

ZSIGA, M ;

BUCHWALD, M ;

RIORDAN, JR ;

TSUI, LC ;

COLLINS, FS .

SCIENCE, 1989, 245 (4922) :1059-1065

[14] Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR [J].

Wainwright, C. E. ;

Elborn, J. S. ;

Ramsey, B. W. ;

Marigowda, G. ;

Huang, X. ;

Cipolli, M. ;

Colombo, C. ;

Davies, J. C. ;

De Boeck, K. ;

Flume, P. A. ;

Konstan, M. W. ;

McColley, S. A. ;

Mccoy, K. ;

McKone, E. F. ;

Munck, A. ;

Ratjen, F. ;

Rowe, S. M. ;

Waltz, D. ;

Boyle, M. P. .

NEW ENGLAND JOURNAL OF MEDICINE, 2015, 373 (03) :220-231

[15] An adaptable standard for protein export from the endoplasmic reticulum [J].

Wiseman, R. Luke ;

Powers, Evan T. ;

Buxbaum, Joel N. ;

Kelly, Jeffery W. ;

Balch, William E. .

CELL, 2007, 131 (04) :809-821

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