-
2
-
-
0000471085
-
Cystic fibrosis of the pancreas and its relation to celiac disease: A clinical and pathologic study
-
Andersen DH. Cystic fibrosis of the pancreas and its relation to celiac disease: a clinical and pathologic study. Am J Dis Child 1938;56: 344-399.
-
(1938)
Am J Dis Child
, vol.56
, pp. 344-399
-
-
Andersen, D.H.1
-
3
-
-
0000536076
-
Das coeliakie-syndrom bei angeborener zystischer pankreasfibromatose und bronchiektasien
-
Fanconi G, Uehlinger E, Knauer C. Das coeliakie-syndrom bei angeborener zystischer pankreasfibromatose und bronchiektasien. Wien Med Wochenschr 1936;86:753-756.
-
(1936)
Wien Med Wochenschr
, vol.86
, pp. 753-756
-
-
Fanconi, G.1
Uehlinger, E.2
Knauer, C.3
-
5
-
-
84991806405
-
CFTR modulators: Shedding light on precision medicine for cystic fibrosis
-
Lopes-Pacheco M. CFTR modulators: shedding light on precision medicine for cystic fibrosis. Front Pharmacol 2016;7:275.
-
(2016)
Front Pharmacol
, vol.7
, pp. 275
-
-
Lopes-Pacheco, M.1
-
6
-
-
84959280942
-
Advancing clinical development pathways for new CFTR modulators in cystic fibrosis
-
Mayer-Hamblett N, Boyle M, VanDevanter D. Advancing clinical development pathways for new CFTR modulators in cystic fibrosis. Thorax 2016;71:454-461.
-
(2016)
Thorax
, vol.71
, pp. 454-461
-
-
Mayer-Hamblett, N.1
Boyle, M.2
VanDevanter, D.3
-
7
-
-
84880044346
-
A new era in the treatment of cystic fibrosis: Correction of the underlying CFTR defect
-
Boyle MP, De Boeck K. A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect. Lancet Respir Med 2013;1: 158-163.
-
(2013)
Lancet Respir Med
, vol.1
, pp. 158-163
-
-
Boyle, M.P.1
De Boeck, K.2
-
8
-
-
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
Grootenhuis, P.D.J.3
Burton, B.4
Cao, D.5
Neuberger, T.6
-
9
-
-
80455162465
-
A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
-
Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, 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
Dřevínek, P.6
-
11
-
-
81755163563
-
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
-
Van Goor F, Hadida S, Grootenhuis PDJ, Burton B, Stack JH, Straley KS, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 2011;108:18843-18848.
-
(2011)
Proc Natl Acad Sci USA
, vol.108
, pp. 18843-18848
-
-
Van Goor, F.1
Hadida, S.2
Grootenhuis, P.D.J.3
Burton, B.4
Stack, J.H.5
Straley, K.S.6
-
12
-
-
33744831154
-
Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
-
Van Goor F, Straley KS, Cao D, González J, Hadida S, Hazlewood A, et al. Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Lung Cell Mol Physiol 2006;290:L1117-L1130.
-
(2006)
Am J Physiol Lung Cell Mol Physiol
, vol.290
, pp. L1117-L1130
-
-
Van Goor, F.1
Straley, K.S.2
Cao, D.3
González, J.4
Hadida, S.5
Hazlewood, A.6
-
13
-
-
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
-
14
-
-
84904410000
-
CFTR modulators for the treatment of cystic fibrosis
-
Pettit RS, Fellner C. CFTR modulators for the treatment of cystic fibrosis. P&T 2014;39:500-511.
-
(2014)
P&T
, vol.39
, pp. 500-511
-
-
Pettit, R.S.1
Fellner, C.2
-
15
-
-
85010871752
-
Use of hyperpolarized helium-3 MRI to assess response to ivacaftor treatment in patients with cystic fibrosis
-
Altes TA, Johnson M, Fidler M, Botfield M, Tustison NJ, Leiva-Salinas C, et al. Use of hyperpolarized helium-3 MRI to assess response to ivacaftor treatment in patients with cystic fibrosis. J Cyst Fibros 2017;16:267-274.
-
(2017)
J Cyst Fibros
, vol.16
, pp. 267-274
-
-
Altes, T.A.1
Johnson, M.2
Fidler, M.3
Botfield, M.4
Tustison, N.J.5
Leiva-Salinas, C.6
-
16
-
-
84995450768
-
Importance of off-label options for treating serious lung diseases
-
O'Sullivan BP. Importance of off-label options for treating serious lung diseases. Ann Am Thorac Soc 2016;13:1879-1880.
-
(2016)
Ann Am Thorac Soc
, vol.13
, pp. 1879-1880
-
-
O'Sullivan, B.P.1
-
17
-
-
84995452405
-
Potential for therapeutic benefit among cystic fibrosis populations excluded from clinical trials or labeling of marketed therapies
-
VanDevanter DR, Heltshe SL, LiPuma JJ. Potential for therapeutic benefit among cystic fibrosis populations excluded from clinical trials or labeling of marketed therapies. Ann Am Thorac Soc 2016;13: 1890-1893.
-
(2016)
Ann Am Thorac Soc
, vol.13
, pp. 1890-1893
-
-
VanDevanter, D.R.1
Heltshe, S.L.2
LiPuma, J.J.3
-
18
-
-
85045252621
-
-
Division of Medical Assistance Programs OHA, 2015 July 30 [updated 2016 Jan 1; accessed 2018 Jan 25]
-
Division of Medical Assistance Programs OHA. Oregon Medicaid Pharmaceutical Services prior authorization criteria: Medicaid PA Criteria, 2015 July 30 [updated 2016 Jan 1; accessed 2018 Jan 25]. Available from: https://www.oregon.gov/oha/healthplan/tools/oregon.
-
Oregon Medicaid Pharmaceutical Services Prior Authorization Criteria: Medicaid PA Criteria
-
-
-
19
-
-
85045280461
-
-
Illinois Department of Healthcare and Family Services Sep [accessed 2017 Sep 1]
-
Illinois Department of Healthcare and Family Services. Orkambi (lumacaftor-ivacaftor tablet): prior authorization criteria. 2015 Sep [accessed 2017 Sep 1]. Available from: http://www.illinois.gov/hfs/sitecollectiondocuments/orkambicriteria.pdf.
-
(2015)
Orkambi (Lumacaftor-ivacaftor Tablet): Prior Authorization Criteria
-
-
-
20
-
-
85045265631
-
-
State of California - Health and Human Services Agency DoHCS, N.L. 06-0915. Sep 4 [accessed 2017 Sep 1]
-
State of California - Health and Human Services Agency DoHCS. Lumacaftor/ivacaftor (Orkambi). N.L. 06-0915. 2015 Sep 4 [accessed 2017 Sep 1]. Available from: http://www.dhcs.ca.gov/services/ccs/documents/ccsnl060915.pdf.
-
(2015)
Lumacaftor/ivacaftor (Orkambi)
-
-
-
21
-
-
84985864585
-
The high cost of prescription drugs in the United States: Origins and prospects for reform
-
Kesselheim AS, Avorn J, Sarpatwari A. The high cost of prescription drugs in the United States: origins and prospects for reform. JAMA 2016;316:858-871.
-
(2016)
JAMA
, vol.316
, pp. 858-871
-
-
Kesselheim, A.S.1
Avorn, J.2
Sarpatwari, A.3
-
22
-
-
85010338550
-
Diagnosis of cystic fibrosis: Consensus guidelines from the Cystic Fibrosis Foundation
-
e11
-
Farrell PM, White TB, Ren CL, Hempstead SE, Accurso F, Derichs N, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr 2017;181:S4-S15.e11.
-
(2017)
J Pediatr
, vol.181
, pp. S4-S15
-
-
Farrell, P.M.1
White, T.B.2
Ren, C.L.3
Hempstead, S.E.4
Accurso, F.5
Derichs, N.6
-
23
-
-
33748305617
-
An official ATS statement: Grading the quality of evidence and strength of recommendations in ATS guidelines and recommendations
-
Schünemann HJ, Jaeschke R, Cook DJ, Bria WF, El-Solh AA, Ernst A, et al.; ATS Documents Development and Implementation Committee. An official ATS statement: grading the quality of evidence and strength of recommendations in ATS guidelines and recommendations. Am J Respir Crit Care Med 2006;174:605-614.
-
(2006)
Am J Respir Crit Care Med
, vol.174
, pp. 605-614
-
-
Schünemann, H.J.1
Jaeschke, R.2
Cook, D.J.3
Bria, W.F.4
El-Solh, A.A.5
Ernst, A.6
-
24
-
-
84875829440
-
Cystic fibrosis pulmonary guidelines: Chronic medications for maintenance of lung health
-
Mogayzel PJ Jr, Naureckas ET, Robinson KA, Mueller G, Hadjiliadis D, Hoag JB, et al.; Pulmonary Clinical Practice Guidelines Committee. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2013;187: 680-689.
-
(2013)
Am J Respir Crit Care Med
, vol.187
, pp. 680-689
-
-
Mogayzel, P.J.1
Naureckas, E.T.2
Robinson, K.A.3
Mueller, G.4
Hadjiliadis, D.5
Hoag, J.B.6
-
25
-
-
84962511867
-
Clinical practice guidelines from the cystic fibrosis foundation for preschoolers with cystic fibrosis
-
Lahiri T, Hempstead SE, Brady C, Cannon CL, Clark K, Condren ME, et al. Clinical practice guidelines from the cystic fibrosis foundation for preschoolers with cystic fibrosis. Pediatrics 2016;137:e20151784.
-
(2016)
Pediatrics
, vol.137
, pp. e20151784
-
-
Lahiri, T.1
Hempstead, S.E.2
Brady, C.3
Cannon, C.L.4
Clark, K.5
Condren, M.E.6
-
26
-
-
84890435909
-
Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function
-
Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros 2014;13:29-36.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 29-36
-
-
Van Goor, F.1
Yu, H.2
Burton, B.3
Hoffman, B.J.4
-
27
-
-
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
-
28
-
-
84911493956
-
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
-
De Boeck K, Munck A, Walker S, Faro A, Hiatt P, Gilmartin G, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros 2014;13:674-680.
-
(2014)
J Cyst Fibros
, vol.13
, pp. 674-680
-
-
De Boeck, K.1
Munck, A.2
Walker, S.3
Faro, A.4
Hiatt, P.5
Gilmartin, G.6
-
29
-
-
0242565793
-
Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis
-
Modi AC, Quittner AL. Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J Pediatr Psychol 2003;28:535-545.
-
(2003)
J Pediatr Psychol
, vol.28
, pp. 535-545
-
-
Modi, A.C.1
Quittner, A.L.2
-
30
-
-
0027730638
-
Detection of 98.5% of the mutations in 200 Belgian cystic fibrosis alleles by reverse dot-blot and sequencing of the complete coding region and exon/intron junctions of the CFTR gene
-
Cuppens H, Marynen P, De Boeck C, Cassiman J-J. Detection of 98.5% of the mutations in 200 Belgian cystic fibrosis alleles by reverse dot-blot and sequencing of the complete coding region and exon/intron junctions of the CFTR gene. Genomics 1993;18: 693-697.
-
(1993)
Genomics
, vol.18
, pp. 693-697
-
-
Cuppens, H.1
Marynen, P.2
De Boeck, C.3
Cassiman, J.-J.4
-
31
-
-
0027521663
-
A mutation in CFTR produces different phenotypes depending on chromosomal background
-
Kiesewetter S, Macek M Jr, Davis C, Curristin SM, Chu CS, Graham C, et al. A mutation in CFTR produces different phenotypes depending on chromosomal background. Nat Genet 1993;5:274-278.
-
(1993)
Nat Genet
, vol.5
, pp. 274-278
-
-
Kiesewetter, S.1
Macek, M.2
Davis, C.3
Curristin, S.M.4
Chu, C.S.5
Graham, C.6
-
32
-
-
0027408231
-
Mutations in CFTR associated with mild-disease-form Cl2 channels with altered pore properties
-
Sheppard DN, Rich DP, Ostedgaard LS, Gregory RJ, Smith AE, Welsh MJ. Mutations in CFTR associated with mild-disease-form Cl2 channels with altered pore properties. Nature 1993;362:160-164.
-
(1993)
Nature
, vol.362
, pp. 160-164
-
-
Sheppard, D.N.1
Rich, D.P.2
Ostedgaard, L.S.3
Gregory, R.J.4
Smith, A.E.5
Welsh, M.J.6
-
33
-
-
84943138352
-
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, et al.; VX11-770-110 (KONDUCT) Study Group. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med 2015;3:524-533.
-
(2015)
Lancet Respir Med
, vol.3
, pp. 524-533
-
-
Moss, R.B.1
Flume, P.A.2
Elborn, J.S.3
Cooke, J.4
Rowe, S.M.5
McColley, S.A.6
-
34
-
-
0027380236
-
The delta F508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane: Determination of functional half-lives on transfected cells
-
Lukacs GL, Chang XB, Bear C, Kartner N, Mohamed A, Riordan JR, et al. The delta F508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane: determination of functional half-lives on transfected cells. J Biol Chem 1993;268:21592-21598.
-
(1993)
J Biol Chem
, vol.268
, pp. 21592-21598
-
-
Lukacs, G.L.1
Chang, X.B.2
Bear, C.3
Kartner, N.4
Mohamed, A.5
Riordan, J.R.6
-
35
-
-
84904002908
-
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, et al.; VX09-809-102 Study Group. 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. Lancet Respir Med 2014;2: 527-538.
-
(2014)
Lancet Respir Med
, vol.2
, pp. 527-538
-
-
Boyle, M.P.1
Bell, S.C.2
Konstan, M.W.3
McColley, S.A.4
Rowe, S.M.5
Rietschel, E.6
-
36
-
-
84994726301
-
Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: A pooled analysis
-
Elborn JS, Ramsey BW, Boyle MP, Konstan MW, Huang X, Marigowda G, et al.; VX-809 TRAFFIC and TRANSPORT study groups. Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis. Lancet Respir Med 2016;4: 617-626.
-
(2016)
Lancet Respir Med
, vol.4
, pp. 617-626
-
-
Elborn, J.S.1
Ramsey, B.W.2
Boyle, M.P.3
Konstan, M.W.4
Huang, X.5
Marigowda, G.6
-
37
-
-
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
-
38
-
-
85015033159
-
Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease
-
Hubert D, Chiron R, Camara B, Grenet D, Prévotat A, Bassinet L, et al. Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease. J Cyst Fibros 2017;16:388-391.
-
(2017)
J Cyst Fibros
, vol.16
, pp. 388-391
-
-
Hubert, D.1
Chiron, R.2
Camara, B.3
Grenet, D.4
Prévotat, A.5
Bassinet, L.6
-
39
-
-
34548278908
-
Power considerations for studies of lung function in cystic fibrosis
-
Corey M. Power considerations for studies of lung function in cystic fibrosis. Proc Am Thorac Soc 2007;4:334-337.
-
(2007)
Proc Am Thorac Soc
, vol.4
, pp. 334-337
-
-
Corey, M.1
-
40
-
-
0031021662
-
Identifying treatments that halt progression of pulmonary disease in cystic fibrosis
-
Davis PB, Byard PJ, Konstan MW. Identifying treatments that halt progression of pulmonary disease in cystic fibrosis. Pediatr Res 1997;41:161-165.
-
(1997)
Pediatr Res
, vol.41
, pp. 161-165
-
-
Davis, P.B.1
Byard, P.J.2
Konstan, M.W.3
-
41
-
-
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
-
42
-
-
84880887459
-
An official American Thoracic Society workshop report: Optimal lung function tests for monitoring cystic fibrosis, bronchopulmonary dysplasia, and recurrent wheezing in children less than 6 years of age
-
Rosenfeld M, Allen J, Arets BH, Aurora P, Beydon N, Calogero C, et al.; American Thoracic Society Assembly on Pediatrics Working Group on Infant and Preschool Lung Function Testing. An official American Thoracic Society workshop report: optimal lung function tests for monitoring cystic fibrosis, bronchopulmonary dysplasia, and recurrent wheezing in children less than 6 years of age. Ann Am Thorac Soc 2013;10:S1-S11.
-
(2013)
Ann Am Thorac Soc
, vol.10
, pp. S1-S11
-
-
Rosenfeld, M.1
Allen, J.2
Arets, B.H.3
Aurora, P.4
Beydon, N.5
Calogero, C.6
-
43
-
-
79952504576
-
Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosi
-
Aurora P, Stanojevic S, Wade A, Oliver C, Kozlowska W, Lum S, et al.; London Cystic Fibrosis Collaboration. Lung clearance index at 4 years predicts subsequent lung function in children with cystic fibrosis. Am J Respir Crit Care Med 2011;183:752-758.
-
(2011)
Am J Respir Crit Care Med
, vol.183
, pp. 752-758
-
-
Aurora, P.1
Stanojevic, S.2
Wade, A.3
Oliver, C.4
Kozlowska, W.5
Lum, S.6
-
44
-
-
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
|