-
1
-
-
84897399962
-
Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: A systematic review and cost-effectiveness analysis
-
Whiting P, Al M, Burgers L, Westwood M, Ryder S, Hoogendoorn M, et al. Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis. Health Technol Assess 2014; 18:1-106.
-
(2014)
Health Technol Assess
, vol.18
, pp. 1-106
-
-
Whiting, P.1
Al, M.2
Burgers, L.3
Westwood, M.4
Ryder, S.5
Hoogendoorn, M.6
-
3
-
-
85006647048
-
-
Cystic Fibrosis Foundation. Cystic fibrosis foundation, overview; 2015 [Accessed 24 March 2016]
-
Cystic Fibrosis Foundation. Cystic fibrosis foundation, overview; 2015. Available at: http://www.cff.org/AboutCF/. [Accessed 24 March 2016].
-
-
-
-
4
-
-
84874726028
-
Targeting a genetic defect: Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis
-
2103
-
Derichs N. Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis. Eur Respir Rev 2103; 22:58-65.
-
Eur Respir Rev
, vol.22
, pp. 58-65
-
-
Derichs, N.1
-
6
-
-
85006644958
-
-
Committee for Medicinal Products for Human Use. Kalydeco: assessment report. London, UK: European Medicines Agency; 2012
-
Committee for Medicinal Products for Human Use. Kalydeco: assessment report. London, UK: European Medicines Agency; 2012.
-
-
-
-
7
-
-
84964067974
-
A safety evaluation of ivacaftor for the treatment of cystic fibrosis
-
McColley SA. A safety evaluation of ivacaftor for the treatment of cystic fibrosis. Expert Opin Drug Saf 2016; 15:709-715.
-
(2016)
Expert Opin Drug Saf
, vol.15
, pp. 709-715
-
-
McColley, S.A.1
-
8
-
-
84907054376
-
Ivacaftor for patients with cystic fibrosis
-
Wainwright CE. Ivacaftor for patients with cystic fibrosis. Expert Rev Respir Med 2014; 8:533-538.
-
(2014)
Expert Rev Respir Med
, vol.8
, pp. 533-538
-
-
Wainwright, C.E.1
-
9
-
-
84919770501
-
Clinical drug-drug interaction assessment of ivacaftor as a potential inhibitor of cytochrome P450 and P-glycoprotein
-
Robertson SM, Luo X, Dubey N, Li C, Chavan AB, Gilmartin GS, et al. Clinical drug-drug interaction assessment of ivacaftor as a potential inhibitor of cytochrome P450 and P-glycoprotein. J Clin Pharmacol 2015; 55:56-62.
-
(2015)
J Clin Pharmacol
, vol.55
, pp. 56-62
-
-
Robertson, S.M.1
Luo, X.2
Dubey, N.3
Li, C.4
Chavan, A.B.5
Gilmartin, G.S.6
-
10
-
-
85006671187
-
-
Committee for Medicinal Products for Human Use. Kalydeco: assessment report. London, UK: European Medicines Agency; 2014
-
Committee for Medicinal Products for Human Use. Kalydeco: assessment report. London, UK: European Medicines Agency; 2014.
-
-
-
-
11
-
-
84949210854
-
Lumacaftor alone and combined with ivacaftor: Preclinical and clinical trial experience of F508del CFTR correction
-
Brewington JJ, McPhail GL, Clancy JP. Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction. Expert Rev Respir Med 2016; 10:5-17.
-
(2016)
Expert Rev Respir Med
, vol.10
, pp. 5-17
-
-
Brewington, J.J.1
McPhail, G.L.2
Clancy, J.P.3
-
12
-
-
78549279173
-
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, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med 2010; 363:1991-2003.
-
(2010)
N Engl J Med
, vol.363
, pp. 1991-2003
-
-
Accurso, F.J.1
Rowe, S.M.2
Clancy, J.P.3
Boyle, M.P.4
Dunitz, J.M.5
Durie, P.R.6
-
13
-
-
7444251037
-
A clinical perspective of cystic fibrosis and new genetic findings: Relationship of CFTR mutations to genotype-phenotype manifestations
-
Kulczycki LL, Kostuch M, Bellanti JA. A clinical perspective of cystic fibrosis and new genetic findings: relationship of CFTR mutations to genotype-phenotype manifestations. Am J Med Genet A 2003; 116A:262-267.
-
(2003)
Am J Med Genet A
, vol.116 A
, pp. 262-267
-
-
Kulczycki, L.L.1
Kostuch, M.2
Bellanti, J.A.3
-
14
-
-
84942412001
-
The future of CFTR modulating therapies for cystic fibrosis
-
Davies JC. The future of CFTR modulating therapies for cystic fibrosis. Curr Opin Pulm Med 2015; 21:579-584.
-
(2015)
Curr Opin Pulm Med
, vol.21
, pp. 579-584
-
-
Davies, J.C.1
-
15
-
-
84875048537
-
Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle
-
Jih KY, Hwang T-C. Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle. Proc Natl Acad Sci USA 2013; 110:4404-4409.
-
(2013)
Proc Natl Acad Sci USA
, vol.110
, pp. 4404-4409
-
-
Jih, K.Y.1
Hwang, T.-C.2
-
16
-
-
84926090443
-
Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels
-
Kopeikin Z, Yuksek Z, Yang HY, Bompadre SG. Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels. J Cyst Fibros 2014; 13:508-514.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 508-514
-
-
Kopeikin, Z.1
Yuksek, Z.2
Yang, H.Y.3
Bompadre, S.G.4
-
17
-
-
84862776940
-
Ivacaftor potentiation of multiple CFTR channels with gating mutations
-
Yu H, Burton B, Huang CJ, Worley J, Cao D, Johnson JP Jr, et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 2012; 11:237-245.
-
(2012)
J Cyst Fibros
, vol.11
, pp. 237-245
-
-
Yu, H.1
Burton, B.2
Huang, C.J.3
Worley, J.4
Cao, D.5
Johnson, J.P.6
-
18
-
-
84885071273
-
Assessment of clinical response to with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: A randomised controlled trial
-
Davies J, Sheridan H, Bell N, Cunningham S, Davies SD, Elborn JS, et al. Assessment of clinical response to with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med 2013; 1:630-638.
-
(2013)
Lancet Respir Med
, vol.1
, pp. 630-638
-
-
Davies, J.1
Sheridan, H.2
Bell, N.3
Cunningham, S.4
Davies, S.D.5
Elborn, J.S.6
-
19
-
-
80455162465
-
A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
-
Ramsey BW, Davies J, McElvaney NG, Tulis E, Bell SC, Dřevínek P, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365:1663-1672.
-
(2011)
N Engl J Med
, vol.365
, pp. 1663-1672
-
-
Ramsey, B.W.1
Davies, J.2
McElvaney, N.G.3
Tulis, E.4
Bell, S.C.5
Dřevínek, P.6
-
20
-
-
84871981271
-
Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: The ivacaftor experience
-
Durmowicz AG, Witzmann KA, Rosebraugh CJ, Chowdhury BA. Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience. Chest 2013; 143:14-18.
-
(2013)
Chest
, vol.143
, pp. 14-18
-
-
Durmowicz, A.G.1
Witzmann, K.A.2
Rosebraugh, C.J.3
Chowdhury, B.A.4
-
21
-
-
84901009642
-
Sweat chloride is not a useful marker of clinical response to ivacaftor
-
Barry PJ, Jones AM, Webb AK, Horsley AR. Sweat chloride is not a useful marker of clinical response to ivacaftor. Thorax 2014; 69:586-587.
-
(2014)
Thorax
, vol.69
, pp. 586-587
-
-
Barry, P.J.1
Jones, A.M.2
Webb, A.K.3
Horsley, A.R.4
-
22
-
-
84895072501
-
Sweat chloride as a biomarker of CFTR activity: Proof of concept and ivacaftor clinical trial data
-
Accurso FJ, van Goor F, Zha J, Stone AJ, Dong Q, Ordonez CL, et al. Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. J Cyst Fibros 2014; 13:139-147.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 139-147
-
-
Accurso, F.J.1
Van Goor, F.2
Zha, J.3
Stone, A.J.4
Dong, Q.5
Ordonez, C.L.6
-
23
-
-
73249114731
-
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
-
Van Goor F, Hadida S, Grootenhuis PDJ, Burton B, Cao D, Neuberger T, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA 2009; 106:18825-18830.
-
(2009)
Proc Natl Acad Sci USA
, vol.106
, pp. 18825-18830
-
-
Van Goor, F.1
Hadida, S.2
Pdj, G.3
Burton, B.4
Cao, D.5
Neuberger, T.6
-
24
-
-
84938677873
-
Pharmaceutical approval update
-
Gohil K. Pharmaceutical approval update. P T 2015; 40:567-568.
-
(2015)
P T
, vol.40
, pp. 567-568
-
-
Gohil, K.1
-
25
-
-
79958746554
-
Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment
-
Rogan MP, Stoltz DA, Hornick DB. Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment. Chest 2011; 139:1480-1490.
-
(2011)
Chest
, vol.139
, pp. 1480-1490
-
-
Rogan, M.P.1
Stoltz, D.A.2
Hornick, D.B.3
-
26
-
-
84885022205
-
Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene
-
Sosnay PR, Siklosi KR, van Goor F, Kaniecki K, Yu H, Sharma N, et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet 2013; 45:1160-1167.
-
(2013)
Nat Genet
, vol.45
, pp. 1160-1167
-
-
Sosnay, P.R.1
Siklosi, K.R.2
Van Goor, F.3
Kaniecki, K.4
Yu, H.5
Sharma, N.6
-
27
-
-
79953212970
-
ACOG Committee Opinion No. 486: Update on carrier screening for cystic fibrosis
-
American College of Obstetricians and Gynecologists Committee on Genetics. ACOG Committee Opinion No. 486: update on carrier screening for cystic fibrosis. Obstet Gynecol 2011; 117:1028-1031.
-
(2011)
Obstet Gynecol
, vol.117
, pp. 1028-1031
-
-
-
28
-
-
84941623631
-
The role of ivacaftor in severe cystic fibrosis in a patient with the R117H mutation
-
Ronan NJ, Fleming C, O'Callaghan G, Maher MM, Murphy DM, Plant BJ. The role of ivacaftor in severe cystic fibrosis in a patient with the R117H mutation. Chest 2015; 148:e72-e75.
-
(2015)
Chest
, vol.148
, pp. e72-e75
-
-
Ronan, N.J.1
Fleming, C.2
O'Callaghan, G.3
Maher, M.M.4
Murphy, D.M.5
Plant, B.J.6
|