Format
Sort by

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 143

1.

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.

Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordoñez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW.

Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8.

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.

Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group.

Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

PMID:
24973281
3.

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

Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group.

N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.

4.

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1.

Ren HY, Grove DE, De La Rosa O, Houck SA, Sopha P, Van Goor F, Hoffman BJ, Cyr DM.

Mol Biol Cell. 2013 Oct;24(19):3016-24. doi: 10.1091/mbc.E13-05-0240. Epub 2013 Aug 7.

5.

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.

Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordoñez CL, Campbell PW, Ashlock MA, Ramsey BW.

N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.

6.

Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation.

Flume PA, Liou TG, Borowitz DS, Li H, Yen K, Ordoñez CL, Geller DE; VX 08-770-104 Study Group.

Chest. 2012 Sep;142(3):718-24.

7.

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.

Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu PA.

Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8. doi: 10.1073/pnas.1105787108. Epub 2011 Oct 5.

8.

Cystic fibrosis transmembrane conductance regulator-modifying medications: the future of cystic fibrosis treatment.

Pettit RS.

Ann Pharmacother. 2012 Jul-Aug;46(7-8):1065-75. doi: 10.1345/aph.1R076. Epub 2012 Jun 26. Review.

PMID:
22739718
9.

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis.

Awatade NT, Uliyakina I, Farinha CM, Clarke LA, Mendes K, Solé A, Pastor J, Ramos MM, Amaral MD.

EBioMedicine. 2014 Dec 17;2(2):147-53. doi: 10.1016/j.ebiom.2014.12.005. eCollection 2015 Feb.

10.

Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation.

Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, Mainz JG, Rodriguez S, Li H, Yen K, Ordoñez CL, Ahrens R; VX08-770-103 (ENVISION) Study Group.

Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25. doi: 10.1164/rccm.201301-0153OC.

11.

Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation.

De Stefano D, Villella VR, Esposito S, Tosco A, Sepe A, De Gregorio F, Salvadori L, Grassia R, Leone CA, De Rosa G, Maiuri MC, Pettoello-Mantovani M, Guido S, Bossi A, Zolin A, Venerando A, Pinna LA, Mehta A, Bona G, Kroemer G, Maiuri L, Raia V.

Autophagy. 2014;10(11):2053-74. doi: 10.4161/15548627.2014.973737.

12.

A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.

Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordoñez C, Elborn JS; VX08-770-102 Study Group.

N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.

13.

Pseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells.

Stanton BA, Coutermarsh B, Barnaby R, Hogan D.

PLoS One. 2015 May 27;10(5):e0127742. doi: 10.1371/journal.pone.0127742. eCollection 2015.

14.

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels.

Kopeikin Z, Yuksek Z, Yang HY, Bompadre SG.

J Cyst Fibros. 2014 Sep;13(5):508-14. doi: 10.1016/j.jcf.2014.04.003. Epub 2014 May 3.

PMID:
24796242
15.

VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface.

Eckford PD, Ramjeesingh M, Molinski S, Pasyk S, Dekkers JF, Li C, Ahmadi S, Ip W, Chung TE, Du K, Yeger H, Beekman J, Gonska T, Bear CE.

Chem Biol. 2014 May 22;21(5):666-78. doi: 10.1016/j.chembiol.2014.02.021. Epub 2014 Apr 10.

16.

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial.

Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group.

Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

17.

Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.

Phuan PW, Veit G, Tan J, Roldan A, Finkbeiner WE, Lukacs GL, Verkman AS.

Mol Pharmacol. 2014 Jul;86(1):42-51. doi: 10.1124/mol.114.092478. Epub 2014 Apr 15.

18.

Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction.

Brewington JJ, McPhail GL, Clancy JP.

Expert Rev Respir Med. 2016;10(1):5-17. doi: 10.1586/17476348.2016.1122527. Epub 2015 Dec 9.

PMID:
26581802
19.

Diversity of the basic defect of homozygous CFTR mutation genotypes in humans.

Stanke F, Ballmann M, Bronsveld I, Dörk T, Gallati S, Laabs U, Derichs N, Ritzka M, Posselt HG, Harms HK, Griese M, Blau H, Mastella G, Bijman J, Veeze H, Tümmler B.

J Med Genet. 2008 Jan;45(1):47-54. doi: 10.1136/jmg.2007.053561.

20.

A double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis.

Knowles MR, Noone PG, Hohneker K, Johnson LG, Boucher RC, Efthimiou J, Crawford C, Brown R, Schwartzbach C, Pearlman R.

Hum Gene Ther. 1998 Jan 20;9(2):249-69.

PMID:
9472784
Items per page

Supplemental Content

Write to the Help Desk