-
1
-
-
85048067969
-
Effects of lumacaftor-ivacaftor therapy on Cystic Fibrosis Transmembrane Conductance Regulator function in Phe508del homozygous patients with cystic fibrosis
-
Graeber SY, Dopfer C, Naehrlich L, Gyulumyan L, Scheuermann H, Hirtz S, et al. Effects of lumacaftor-ivacaftor therapy on Cystic Fibrosis Transmembrane Conductance Regulator function in Phe508del homozygous patients with cystic fibrosis. Am J Respir Crit Care Med 2018;197:1433-1442.
-
(2018)
Am J Respir Crit Care Med
, vol.197
, pp. 1433-1442
-
-
Graeber, S.Y.1
Dopfer, C.2
Naehrlich, L.3
Gyulumyan, L.4
Scheuermann, H.5
Hirtz, S.6
-
2
-
-
84937035647
-
Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR
-
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, et al.; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015;373:220-231.
-
(2015)
N Engl J Med
, vol.373
, pp. 220-231
-
-
Wainwright, C.E.1
Elborn, J.S.2
Ramsey, B.W.3
Marigowda, G.4
Huang, X.5
Cipolli, M.6
-
3
-
-
84949035982
-
Intestinal current measurements detect activation of mutant CFTR in patients with cystic fibrosis with the G551D mutation treated with ivacaftor
-
Graeber SY, Hug MJ, Sommerburg O, Hirtz S, Hentschel J, Heinzmann A, et al. Intestinal current measurements detect activation of mutant CFTR in patients with cystic fibrosis with the G551D mutation treated with ivacaftor. Am J Respir Crit Care Med 2015;192:1252-1255.
-
(2015)
Am J Respir Crit Care Med
, vol.192
, pp. 1252-1255
-
-
Graeber, S.Y.1
Hug, M.J.2
Sommerburg, O.3
Hirtz, S.4
Hentschel, J.5
Heinzmann, A.6
-
4
-
-
80052340084
-
Nasal potential difference measurements to assess CFTR ion channel activity
-
Rowe SM, Clancy JP, Wilschanski M. Nasal potential difference measurements to assess CFTR ion channel activity. Methods Mol Biol 2011;741:69-86.
-
(2011)
Methods Mol Biol
, vol.741
, pp. 69-86
-
-
Rowe, S.M.1
Clancy, J.P.2
Wilschanski, M.3
-
5
-
-
84880774140
-
Optimizing nasal potential difference analysis for CFTR modulator development: Assessment of ivacaftor in CF subjects with the G551D-CFTR mutation
-
Rowe SM, Liu B, Hill A, Hathorne H, Cohen M, Beamer JR, et al.; VX06-770-101 Study Group. Optimizing nasal potential difference analysis for CFTR modulator development: assessment of ivacaftor in CF subjects with the G551D-CFTR mutation. PLoS One 2013;8: e66955.
-
(2013)
PLoS One
, vol.8
-
-
Rowe, S.M.1
Liu, B.2
Hill, A.3
Hathorne, H.4
Cohen, M.5
Beamer, J.R.6
-
6
-
-
84961221214
-
Variability of sweat chloride concentration in subjects with cystic fibrosis and G551D mutations
-
Vermeulen F, Le Camus C, Davies JC, Bilton D, Milenković D, De Boeck K. Variability of sweat chloride concentration in subjects with cystic fibrosis and G551D mutations. J Cyst Fibros 2017;16: 36-40.
-
(2017)
J Cyst Fibros
, vol.16
, pp. 36-40
-
-
Vermeulen, F.1
Le Camus, C.2
Davies, J.C.3
Bilton, D.4
Milenković, D.5
De Boeck, K.6
-
7
-
-
5144232620
-
CFTR Cl- Channel function in native human colon correlates with the genotype and phenotype in cystic fibrosis
-
Hirtz S, Gonska T, Seydewitz HH, Thomas J, Greiner P, Kuehr J, et al. CFTR Cl- channel function in native human colon correlates with the genotype and phenotype in cystic fibrosis. Gastroenterology 2004;127:1085-1095.
-
(2004)
Gastroenterology
, vol.127
, pp. 1085-1095
-
-
Hirtz, S.1
Gonska, T.2
Seydewitz, H.H.3
Thomas, J.4
Greiner, P.5
Kuehr, J.6
-
8
-
-
85017338127
-
Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis and homozygous for F508del-CFTR
-
Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld M; VX13-809-011 Part B Investigator Group. Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis and homozygous for F508del-CFTR. Am J Respir Crit Care Med 2017;195:912-920.
-
(2017)
Am J Respir Crit Care Med
, vol.195
, pp. 912-920
-
-
Milla, C.E.1
Ratjen, F.2
Marigowda, G.3
Liu, F.4
Waltz, D.5
Rosenfeld, M.6
-
9
-
-
85020383955
-
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: A randomised, placebo-controlled phase 3 trial
-
Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, et al.; VX14-809-109 investigator group. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med 2017;5:557-567.
-
(2017)
Lancet Respir Med
, vol.5
, pp. 557-567
-
-
Ratjen, F.1
Hug, C.2
Marigowda, G.3
Tian, S.4
Huang, X.5
Stanojevic, S.6
-
10
-
-
85023598529
-
Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting
-
Hubert D, Dehillotte C, Munck A, David V, Baek J, Mely L, et al. Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting. J Cyst Fibros 2018; 17:89-95.
-
(2018)
J Cyst Fibros
, vol.17
, pp. 89-95
-
-
Hubert, D.1
Dehillotte, C.2
Munck, A.3
David, V.4
Baek, J.5
Mely, L.6
-
11
-
-
84943328860
-
Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data
-
Sawicki GS, McKone EF, Pasta DJ, Millar SJ, Wagener JS, Johnson CA, et al. Sustained benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med 2015;192:836-842.
-
(2015)
Am J Respir Crit Care Med
, vol.192
, pp. 836-842
-
-
Sawicki, G.S.1
McKone, E.F.2
Pasta, D.J.3
Millar, S.J.4
Wagener, J.S.5
Johnson, C.A.6
-
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
-
-
0034234755
-
Residual chloride secretion in intestinal tissue of deltaF508 homozygous twins and siblings with cystic fibrosis
-
Bronsveld I, Mekus F, Bijman J, Ballmann M, Greipel J, Hundrieser J, et al.; The European CF Twin and Sibling Study Consortium. Residual chloride secretion in intestinal tissue of deltaF508 homozygous twins and siblings with cystic fibrosis. Gastroenterology 2000; 119:32-40.
-
(2000)
Gastroenterology
, vol.119
, pp. 32-40
-
-
Bronsveld, I.1
Mekus, F.2
Bijman, J.3
Ballmann, M.4
Greipel, J.5
Hundrieser, J.6
-
14
-
-
85008221944
-
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): A phase 3, extension study
-
Konstan MW, McKone EF, Moss RB, Marigowda G, Tian S, Waltz D, et al. Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. Lancet Respir Med 2017; 5:107-118.
-
(2017)
Lancet Respir Med
, vol.5
, pp. 107-118
-
-
Konstan, M.W.1
McKone, E.F.2
Moss, R.B.3
Marigowda, G.4
Tian, S.5
Waltz, D.6
-
15
-
-
85034625790
-
-
Washington, DC: U.S. Food and Drug Administration; accessed 2018 Jan 24.
-
FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis. Washington, DC: U.S. Food and Drug Administration; 2017 [accessed 2018 Jan 24]. Available from: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.
-
(2017)
FDA Expands Approved Use of Kalydeco to Treat Additional Mutations of Cystic Fibrosis
-
-
-
16
-
-
85039797009
-
The U.S. Food and Drug Administration's experience with ivacaftor in cystic fibrosis: Establishing efficacy using in vitro data in lieu of a clinical trial
-
Durmowicz AG, Lim R, Rogers H, Rosebraugh CJ, Chowdhury BA. The U.S. Food and Drug Administration's experience with ivacaftor in cystic fibrosis: establishing efficacy using in vitro data in lieu of a clinical trial. Ann Am Thorac Soc 2018;15:1-2.
-
(2018)
Ann Am Thorac Soc
, vol.15
, pp. 1-2
-
-
Durmowicz, A.G.1
Lim, R.2
Rogers, H.3
Rosebraugh, C.J.4
Chowdhury, B.A.5
-
17
-
-
85028719507
-
FDA OKs first in vitro route to expanded approval
-
Kingwell K. FDA OKs first in vitro route to expanded approval. Nat Rev Drug Discov 2017;16:591-592.
-
(2017)
Nat Rev Drug Discov
, vol.16
, pp. 591-592
-
-
Kingwell, K.1
-
18
-
-
85030449367
-
Recent progress in translational cystic fibrosis research using precision medicine strategies
-
Cholon DM, Gentzsch M. Recent progress in translational cystic fibrosis research using precision medicine strategies. J Cyst Fibros 2018;17(2S):S52-S60.
-
(2018)
J Cyst Fibros
, vol.17
, Issue.2 S
, pp. S52-S60
-
-
Cholon, D.M.1
Gentzsch, M.2
-
19
-
-
84998693423
-
Cystic fibrosis biomarker: Commentary
-
Durmowicz AG. Cystic fibrosis biomarker: commentary. J Cyst Fibros 2016;15:712-713.
-
(2016)
J Cyst Fibros
, vol.15
, pp. 712-713
-
-
Durmowicz, A.G.1
|