-
1
-
-
0037075272
-
Pure red-cell aplasia and antierythropoietin antibodies in patients treated with recombinant erythropoietin
-
CASADEVALL N, NATAF J, VIRON B, et al: Pure red-cell aplasia and antierythropoietin antibodies in patients treated with recombinant erythropoietin. N Engl J Med 346:469-475, 2002
-
(2002)
N Engl J Med
, vol.346
, pp. 469-475
-
-
Casadevall, N.1
Nataf, J.2
Viron, B.3
-
2
-
-
0027473752
-
Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors
-
Kogenate Previously Untreated Patient Study Group
-
LUSHER JM, ARKIN S, ABILDGAARD CF, et al: Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 328:453-459, 1993
-
(1993)
N Engl J Med
, vol.328
, pp. 453-459
-
-
Lusher, J.M.1
Arkin, S.2
Abildgaard, C.F.3
-
3
-
-
0025869216
-
Replacement therapy for inherited enzyme deficiency - Macrophage-targeted glucocerebrosidase for Gaucher's disease
-
BARTON NW, BRADY RO, DAMBROSIA JM, et al: Replacement therapy for inherited enzyme deficiency - Macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 324:1464-1470, 1991
-
(1991)
N Engl J Med
, vol.324
, pp. 1464-1470
-
-
Barton, N.W.1
Brady, R.O.2
Dambrosia, J.M.3
-
4
-
-
0035811624
-
Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry's disease
-
ENG CM, GUFFON N, WILCOX WR, et al: Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry's disease. N Engl J Med 345:9-16, 2001
-
(2001)
N Engl J Med
, vol.345
, pp. 9-16
-
-
Eng, C.M.1
Guffon, N.2
Wilcox, W.R.3
-
5
-
-
0035816007
-
Enzyme replacement therapy in Fabry disease: A randomized controlled trial
-
SCHIFFMANN R, KOPP JB, AUSTIN HA III, et al: Enzyme replacement therapy in Fabry disease: A randomized controlled trial. JAMA 285:2743-2749, 2001.
-
(2001)
JAMA
, vol.285
, pp. 2743-2749
-
-
Schiffmann, R.1
Kopp, J.B.2
Austin III, H.A.3
-
6
-
-
0034729963
-
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients
-
VAN DEN HOUT H, REUSER AJ, VULTO AG, et al: Recombinant human alpha-glucosidase from rabbit milk in Pompe patients. Lancet 356:397-398, 2000
-
(2000)
Lancet
, vol.356
, pp. 397-398
-
-
Van Den Hout, H.1
Reuser, A.J.2
Vulto, A.G.3
-
7
-
-
0035905889
-
Enzyme-replacement therapy in mucopolysaccharidosis I
-
KAKKIS ED, MUENZER J, TILLER GE, et al: Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med 344:182-188, 2001
-
(2001)
N Engl J Med
, vol.344
, pp. 182-188
-
-
Kakkis, E.D.1
Muenzer, J.2
Tiller, G.E.3
-
8
-
-
0000889058
-
α-galactosidase A deficiency: Fabry disease
-
edited by Scriver CR, Beaudet AL, Sly WS, Valle D, New York, McGraw-Hill
-
DESNICK RJ, IOANNOU YA, ENG ME: α-galactosidase A deficiency: Fabry disease, in The Metabolic and Molecular Bases of Inherited Disease (vol 3), 8th ed, edited by Scriver CR, Beaudet AL, Sly WS, Valle D, New York, McGraw-Hill, 2001, pp 3733-3774
-
(2001)
The Metabolic and Molecular Bases of Inherited Disease (vol 3), 8th Ed.
, vol.3
, pp. 3733-3774
-
-
Desnick, R.J.1
Ioannou, Y.A.2
Eng, M.E.3
-
9
-
-
0036436320
-
Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy
-
THURBERG BL, RENNKE H, COLVIN RB, et al: Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney Int 62:1933-1946, 2002
-
(2002)
Kidney Int
, vol.62
, pp. 1933-1946
-
-
Thurberg, B.L.1
Rennke, H.2
Colvin, R.B.3
-
10
-
-
12144287518
-
Fabry disease defined: Baseline clinical manifestations of 366 patients in the Fabry Outcome Survey
-
MEHTA A, RICCI R, WIDMER U, et al: Fabry disease defined: Baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. Eur J Clin Invest 34:236-242, 2004
-
(2004)
Eur J Clin Invest
, vol.34
, pp. 236-242
-
-
Mehta, A.1
Ricci, R.2
Widmer, U.3
-
11
-
-
0036016096
-
Agalsidase alfa - A preparation for enzyme replacement therapy in Anderson-Fabry disease
-
BECK M: Agalsidase alfa - A preparation for enzyme replacement therapy in Anderson-Fabry disease. Expert Opin Investig Drugs 11:851-858, 2002
-
(2002)
Expert Opin Investig Drugs
, vol.11
, pp. 851-858
-
-
Beck, M.1
-
12
-
-
0035949059
-
Enzyme-replacement therapy for Anderson-Fabry disease
-
PASTORES GM, THADHANI R: Enzyme-replacement therapy for Anderson-Fabry disease. Lancet 358:601-603, 2001
-
(2001)
Lancet
, vol.358
, pp. 601-603
-
-
Pastores, G.M.1
Thadhani, R.2
-
13
-
-
0019464277
-
Differential assay for lysosomal α-galactosidases in human tissues and its application to Fabry's disease
-
MAYES JS, SCHEERER JB, SIFERS RN, et al: Differential assay for lysosomal α-galactosidases in human tissues and its application to Fabry's disease. Clin Chim Acta 112:247-251, 1981
-
(1981)
Clin Chim Acta
, vol.112
, pp. 247-251
-
-
Mayes, J.S.1
Scheerer, J.B.2
Sifers, R.N.3
-
14
-
-
70449158340
-
A simple method for the isolation and purification of total lipids from animal tissues
-
FOLCH J, LEES M, SLOANE-STANLEY GH: A simple method for the isolation and purification of total lipids from animal tissues. J Biol Chem 226:497-509, 1957
-
(1957)
J Biol Chem
, vol.226
, pp. 497-509
-
-
Folch, J.1
Lees, M.2
Sloane-Stanley, G.H.3
-
15
-
-
0033559287
-
Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: Induction of humoral tolerance in seroconverted patients after repeat administration
-
ROSENBERG M, KINGMA W, FITZPATRICK MA, et al: Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: Induction of humoral tolerance in seroconverted patients after repeat administration. Blood 93:2081-2088, 1999
-
(1999)
Blood
, vol.93
, pp. 2081-2088
-
-
Rosenberg, M.1
Kingma, W.2
Fitzpatrick, M.A.3
-
16
-
-
0031290143
-
Management of neutralizing antibody to ceredase in a patient with type 3 Gaucher disease
-
BRADY RO, MURRAY GJ, OLIVER KL, et al: Management of neutralizing antibody to ceredase in a patient with type 3 Gaucher disease. Pediatrics 100:E11, 1997
-
(1997)
Pediatrics
, vol.100
-
-
Brady, R.O.1
Murray, G.J.2
Oliver, K.L.3
-
17
-
-
0142153023
-
Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder
-
BROOKS DA, KAKAVANOS R, HOPWOOD JJ: Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder. Trends Mol Med 9:450-453, 2003
-
(2003)
Trends Mol Med
, vol.9
, pp. 450-453
-
-
Brooks, D.A.1
Kakavanos, R.2
Hopwood, J.J.3
-
18
-
-
85030818390
-
-
FOOD AND DRUG ADMINISTRATION: FDA briefing document Fabrazyme: http://www.fda.gov/ohrms/dockets/ac/03/briefing/3917B1_03_FDA%20backgrounder. pdf. 2002
-
(2002)
FDA Briefing Document Fabrazyme
-
-
-
19
-
-
4644315119
-
-
FOOD AND DRUG ADMINISTRATION: FDA briefing report Replagal: http://www.fda.gov/ohrms/dockets/ac/03/briefing/3917B2'02'FDA-backgrounder.pdf. 2002
-
(2002)
FDA Briefing Report Replagal
-
-
-
20
-
-
0037219559
-
Recombinant enzyme therapy for Fabry disease. Absence of editing of human α-galactosidase A mRNA
-
BLOM D, SPEIJER D, LINTHORST GE, et al: Recombinant enzyme therapy for Fabry disease. Absence of editing of human α-galactosidase A mRNA. Am J Hum Genet 72:23-31, 2003
-
(2003)
Am J Hum Genet
, vol.72
, pp. 23-31
-
-
Blom, D.1
Speijer, D.2
Linthorst, G.E.3
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