-
1
-
-
84939224261
-
The evolution of cystic fibrosis care
-
Pittman JE, Ferkol TW. The evolution of cystic fibrosis care. Chest 2015; 148:533-542.
-
(2015)
Chest
, vol.148
, pp. 533-542
-
-
Pittman, J.E.1
Ferkol, T.W.2
-
3
-
-
84866137956
-
Progress in cystic fibrosis and the CF Therapeutics Development Network
-
Rowe SM, Borowitz DS, Burns JL, Clancy JP, Donaldson SH, Retsch-Bogart G, Sagel SD, Ramsey BW. Progress in cystic fibrosis and the CF Therapeutics Development Network. Thorax 2012;67:882-890.
-
(2012)
Thorax
, vol.67
, pp. 882-890
-
-
Rowe, S.M.1
Borowitz, D.S.2
Burns, J.L.3
Clancy, J.P.4
Donaldson, S.H.5
Retsch-Bogart, G.6
Sagel, S.D.7
Ramsey, B.W.8
-
5
-
-
80455162465
-
A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
-
Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, D?revínek P, Griese M, McKone EF, Wainwright CE, Konstan MW, et al.; VX08-770-102 Study Group. 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
Tullis, E.4
Bell, S.C.5
Drevínek, P.6
Griese, M.7
McKone, E.F.8
Wainwright, C.E.9
Konstan, M.W.10
-
6
-
-
84942267624
-
The $300,000 drug
-
July [accessed 2015 Jul 20]
-
Nocera J. The $300,000 drug. New York Times 2014 July 18 [accessed 2015 Jul 20]. Available from: http://www.nytimes.com/2014/07/19/opinion/joe-nocera-cystic-fibrosis-drug-price.html
-
(2014)
New York Times
, vol.18
-
-
Nocera, J.1
-
7
-
-
84855202429
-
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, et al. 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. Thorax 2012;67:12-18.
-
(2012)
Thorax
, vol.67
, pp. 12-18
-
-
Clancy, J.P.1
Rowe, S.M.2
Accurso, F.J.3
Aitken, M.L.4
Amin, R.S.5
Ashlock, M.A.6
Ballmann, M.7
Boyle, M.P.8
Bronsveld, I.9
Campbell, P.W.10
-
8
-
-
84937035647
-
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, 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
Colombo, C.7
Davies, J.C.8
De Boeck, K.9
Flume, P.A.10
-
9
-
-
77951887288
-
Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: A randomized controlled trial
-
Saiman L, Anstead M, Mayer-Hamblett N, Lands LC, Kloster M, Hocevar-Trnka J, Goss CH, Rose LM, Burns JL, Marshall BC, et al.; AZ0004 Azithromycin Study Group. Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA 2010; 303:1707-1715.
-
(2010)
JAMA
, vol.303
, pp. 1707-1715
-
-
Saiman, L.1
Anstead, M.2
Mayer-Hamblett, N.3
Lands, L.C.4
Kloster, M.5
Hocevar-Trnka, J.6
Goss, C.H.7
Rose, L.M.8
Burns, J.L.9
Marshall, B.C.10
-
10
-
-
84907223425
-
Potentiator ivacaftor abrogates pharmacological correction of DF508 CFTR in cystic fibrosis
-
Cholon DM, Quinney NL, Fulcher ML, Esther CR Jr, Das J, Dokholyan NV, Randell SH, Boucher RC, Gentzsch M. Potentiator ivacaftor abrogates pharmacological correction of DF508 CFTR in cystic fibrosis. Sci Transl Med 2014;6:246ra96.
-
(2014)
Sci Transl Med
, vol.6
, pp. 246ra96
-
-
Cholon, D.M.1
Quinney, N.L.2
Fulcher, M.L.3
Esther, C.R.4
Das, J.5
Dokholyan, N.V.6
Randell, S.H.7
Boucher, R.C.8
Gentzsch, M.9
-
11
-
-
84907211027
-
Some gating potentiators, including VX-770, diminish DF508-CFTR functional expression
-
Veit G, Avramescu RG, Perdomo D, Phuan PW, Bagdany M, Apaja PM, Borot F, Szollosi D, Wu YS, Finkbeiner WE, et al. Some gating potentiators, including VX-770, diminish DF508-CFTR functional expression. Sci Transl Med 2014;6:246ra97.
-
(2014)
Sci Transl Med
, vol.6
, pp. 246ra97
-
-
Veit, G.1
Avramescu, R.G.2
Perdomo, D.3
Phuan, P.W.4
Bagdany, M.5
Apaja, P.M.6
Borot, F.7
Szollosi, D.8
Wu, Y.S.9
Finkbeiner, W.E.10
-
12
-
-
84937053668
-
Another beginning for cystic fibrosis therapy
-
Davis PB. Another beginning for cystic fibrosis therapy. N Engl J Med 2015;373:274-276.
-
(2015)
N Engl J Med
, vol.373
, pp. 274-276
-
-
Davis, P.B.1
-
13
-
-
85020310194
-
Orkambi a new cystic fibrosis drug wins F.D.A. Approval
-
July 2 [accessed 2015 Jul 20]
-
Pollack A. Orkambi, a new cystic fibrosis drug, wins F.D.A. approval. New York Times 2015 July 2 [accessed 2015 Jul 20]. Available from: http://www.nytimes.com/2015/07/03/business/orkambi-a-newcystic-fibrosis-drug-wins-fda-approval.html
-
(2015)
New York Times
-
-
Pollack, A.1
-
14
-
-
84884931356
-
Pricing for orphan drugs: Will the market bear what society cannot?
-
O'Sullivan BP, Orenstein DM, Milla CE. Pricing for orphan drugs: will the market bear what society cannot? JAMA 2013;310:1343-1344.
-
(2013)
JAMA
, vol.310
, pp. 1343-1344
-
-
O'Sullivan, B.P.1
Orenstein, D.M.2
Milla, C.E.3
-
15
-
-
84942242300
-
-
New York Times 2015 January 14 [accessed 2015 Jul 20]
-
Bach PB. Why drugs cost so much. New York Times 2015 January 14 [accessed 2015 Jul 20]. Available from: http://www.nytimes.com/2015/01/15/opinion/why-drugs-cost-so-much.html
-
Why Drugs Cost so Much
-
-
Bach, P.B.1
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