-
1
-
-
0025869216
-
Replacement therapy for inherited enzyme deficiency - macrophage- targeted glucocerebrosidase for Gaucher’s disease
-
Barton, N.W. et al. Replacement therapy for inherited enzyme deficiency - macrophage- targeted glucocerebrosidase for Gaucher’s disease. N. Engl. J. Med., 324, 1464, 1991.
-
(1991)
N. Engl. J. Med
, vol.324
, pp. 1464
-
-
Barton, N.W.1
-
2
-
-
0028883136
-
Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources
-
Grabowski, G.A. et al. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann. Intern. Med., 122, 33, 1995.
-
(1995)
Ann. Intern. Med
, vol.122
, pp. 33
-
-
Grabowski, G.A.1
-
3
-
-
2942588994
-
Enzyme replacement and enhancement therapies for lysosomal diseases
-
Desnick, R.J. Enzyme replacement and enhancement therapies for lysosomal diseases. J. Inherit. Metab. Dis., 27, 385, 2004.
-
(2004)
J. Inherit. Metab. Dis
, vol.27
, pp. 385
-
-
Desnick, R.J.1
-
4
-
-
0027199108
-
Home treatment with intravenous enzyme replacement therapy for Gaucher disease: An international collaborative study of 33 patients
-
Zimran, A. et al. Home treatment with intravenous enzyme replacement therapy for Gaucher disease: an international collaborative study of 33 patients. Blood. 82, 1107, 1993.
-
(1993)
Blood
, vol.82
, pp. 1107
-
-
Zimran, A.1
-
6
-
-
0031444906
-
Use of enzyme replacement therapy for Gaucher disease during pregnancy
-
Elstein, D. et al. Use of enzyme replacement therapy for Gaucher disease during pregnancy. Am. J. Obstet. Gynecol., 177, 1509, 1997.
-
(1997)
Am. J. Obstet. Gynecol
, vol.177
, pp. 1509
-
-
Elstein, D.1
-
7
-
-
1342281753
-
Pregnancies in Gaucher disease: A 5-year study
-
Elstein, Y. et al. Pregnancies in Gaucher disease: a 5-year study. Am. J. Obstet. Gynecol. 190, 435, 2004.
-
(2004)
Am. J. Obstet. Gynecol
, vol.190
, pp. 435
-
-
Elstein, Y.1
-
8
-
-
0033559287
-
Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: Induction of humoral tolerance in seroconverted patients after repeat administration
-
Rosenberg, M. et al. Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration. Blood. 93, 2081, 1999.
-
(1999)
Blood
, vol.93
, pp. 2081
-
-
Rosenberg, M.1
-
9
-
-
17944398567
-
Echocardiographic assessment of pulmonary hypertension in Gaucher’s disease
-
Elstein, D. et al. Echocardiographic assessment of pulmonary hypertension in Gaucher’s disease. Lancet. 351, 1544, 1998.
-
(1998)
Lancet
, vol.351
, pp. 1544
-
-
Elstein, D.1
-
10
-
-
0034892109
-
Lung involvement and enzyme replacement therapy in Gaucher’s disease
-
Goitein, O. et al. Lung involvement and enzyme replacement therapy in Gaucher’s disease. QJM. 94, 407, 2001.
-
(2001)
QJM
, vol.94
, pp. 407
-
-
Goitein, O.1
-
11
-
-
0036399154
-
Pulmonary hypertension in type 1 Gaucher’s disease: Genetic and epigenetic determinants of phenotype and response to therapy
-
Mistry, P.K. et al. Pulmonary hypertension in type 1 Gaucher’s disease: genetic and epigenetic determinants of phenotype and response to therapy. Mol. Genet. Metab., 77, 91, 2002.
-
(2002)
Mol. Genet. Metab
, vol.77
, pp. 91
-
-
Mistry, P.K.1
-
12
-
-
4744358540
-
Gaucher disease type 1: Revised recommendations on evaluations and monitoring for adult patients
-
Weinreb, N.J. et al. Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. Semin. Hematol., 41 (Suppl. 5), 15, 2004.
-
(2004)
Semin. Hematol
, vol.41
, pp. 15
-
-
Weinreb, N.J.1
-
13
-
-
0031841598
-
Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease
-
Elstein, D. et al. Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease. QJM. 91, 483, 1998.
-
(1998)
QJM
, vol.91
, pp. 483
-
-
Elstein, D.1
-
14
-
-
0022647117
-
Effect of splenectomy on destructive bone changes in children with chronic (Type I) Gaucher disease
-
Ashkenazi, A., Zaizov, R., and Matoth, Y. Effect of splenectomy on destructive bone changes in children with chronic (Type I) Gaucher disease. Eur. J. Pediatr., 145, 138, 1986.
-
(1986)
Eur. J. Pediatr
, vol.145
, pp. 138
-
-
Ashkenazi, A.1
Zaizov, R.2
Matoth, Y.3
-
15
-
-
0032829209
-
Is there a correlation between degree of splenomegaly, symptoms and hypersplenism? A study of 218 patients with Gaucher disease
-
Gielchinsky, Y. et al. Is there a correlation between degree of splenomegaly, symptoms and hypersplenism? A study of 218 patients with Gaucher disease. Br. J. Haematol., 106, 812, 1999.
-
(1999)
Br. J. Haematol
, vol.106
, pp. 812
-
-
Gielchinsky, Y.1
-
16
-
-
0035963926
-
Gaucher’s disease
-
Elstein, D. et al. Gaucher’s disease. Lancet. 358, 324, 2001.
-
(2001)
Lancet
, vol.358
, pp. 324
-
-
Elstein, D.1
-
18
-
-
0028359980
-
Low-dose enzyme replacement therapy for Gaucher’s disease: Effects of age, sex, genotype, and clinical features on response to treatment
-
Zimran, A. et al. Low-dose enzyme replacement therapy for Gaucher’s disease: effects of age, sex, genotype, and clinical features on response to treatment. Am. J. Med., 97, 3, 1994.
-
(1994)
Am. J. Med
, vol.97
, pp. 3
-
-
Zimran, A.1
-
19
-
-
0037159549
-
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry
-
Weinreb, N.J. et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. Am. J. Med., 113, 112, 2002.
-
(2002)
Am. J. Med
, vol.113
, pp. 112
-
-
Weinreb, N.J.1
-
20
-
-
0030317958
-
Pulmonary hypertension developing after alglucerase therapy in two patients with type 1 Gaucher disease complicated by the hepatopulmonary syndrome
-
Dawson, A. et al. Pulmonary hypertension developing after alglucerase therapy in two patients with type 1 Gaucher disease complicated by the hepatopulmonary syndrome. Ann. Intern. Med., 125, 901, 1996.
-
(1996)
Ann. Intern. Med
, vol.125
, pp. 901
-
-
Dawson, A.1
-
21
-
-
4744370348
-
Therapeutic goals in the treatment of Gaucher disease
-
Pastores, G.M. et al. Therapeutic goals in the treatment of Gaucher disease. Semin. Hematol., 41 (Suppl. 5), 4, 2004.
-
(2004)
Semin. Hematol
, vol.41
, pp. 4
-
-
Pastores, G.M.1
-
22
-
-
0021253016
-
Immune thrombocytopenia and Gaucher’s disease
-
Lester, T.J. et al. Immune thrombocytopenia and Gaucher’s disease. Am. J. Med., 77, 569, 1984.
-
(1984)
Am. J. Med
, vol.77
, pp. 569
-
-
Lester, T.J.1
-
23
-
-
0026727643
-
Successful treatment of bone marrow failure in Gaucher’s disease with low-dose modified glucocerebrosidase
-
Mistry, P.K. et al. Successful treatment of bone marrow failure in Gaucher’s disease with low-dose modified glucocerebrosidase. QJM. 83, 541, 1992.
-
(1992)
QJM
, vol.83
, pp. 541
-
-
Mistry, P.K.1
-
24
-
-
0032977820
-
Platelet function abnormalities in Gaucher disease patients
-
Gillis, S. et al. Platelet function abnormalities in Gaucher disease patients. Am. J. Hematol., 61, 103, 1999.
-
(1999)
Am. J. Hematol
, vol.61
, pp. 103
-
-
Gillis, S.1
-
25
-
-
24644466574
-
Survey of hematological aspects of Gaucher disease
-
Zimran, A. et al. Survey of hematological aspects of Gaucher disease. Hematology. 10, 151, 2005.
-
(2005)
Hematology
, vol.10
, pp. 151
-
-
Zimran, A.1
-
26
-
-
18844388455
-
Gaucher disease and cancer incidence: A study from the Gaucher registry
-
Rosenbloom, B.E. et al. Gaucher disease and cancer incidence: a study from the Gaucher registry. Blood. 105, 4569, 2005.
-
(2005)
Blood
, vol.105
, pp. 4569
-
-
Rosenbloom, B.E.1
-
27
-
-
18844449947
-
Incidence of malignancies among patients with type I Gaucher disease from a single referral clinic
-
Zimran, A. et al. Incidence of malignancies among patients with type I Gaucher disease from a single referral clinic. Blood Cells Mol. Dis., 34, 197, 2005.
-
(2005)
Blood Cells Mol. Dis
, vol.34
, pp. 197
-
-
Zimran, A.1
-
28
-
-
0024686922
-
Recombinant erythropoietin improves the anemia associated with Gaucher’s disease
-
Rodgers, G.P. and Lessin, L.S. Recombinant erythropoietin improves the anemia associated with Gaucher’s disease. Blood. 73, 2228, 1989.
-
(1989)
Blood
, vol.73
, pp. 2228
-
-
Rodgers, G.P.1
Lessin, L.S.2
-
29
-
-
0036373593
-
Thrombocytosis associated with enzyme replacement therapy in Gaucher disease
-
Dweck. A. et al. Thrombocytosis associated with enzyme replacement therapy in Gaucher disease. Acta Haematol., 108, 94, 2002.
-
(2002)
Acta Haematol
, vol.108
, pp. 94
-
-
Dweck, A.1
-
30
-
-
0027204822
-
Correction of neutrophil chemotaxis defect in patients with Gaucher disease by low-dose enzyme replacement therapy
-
Zimran, A. et al. Correction of neutrophil chemotaxis defect in patients with Gaucher disease by low-dose enzyme replacement therapy. Am. J. Hematol., 43, 69, 1993.
-
(1993)
Am. J. Hematol
, vol.43
, pp. 69
-
-
Zimran, A.1
-
32
-
-
0029155493
-
Enzyme replacement therapy for Gaucher disease: Skeletal responses to macrophage-targeted glucocerebrosidase
-
Rosental, D.I. et al. Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. Pediatrics. 96, 629, 1995.
-
(1995)
Pediatrics
, vol.96
, pp. 629
-
-
Rosental, D.I.1
-
33
-
-
0036073123
-
Response of Gaucher bone disease to enzyme replacement therapy
-
Poll, L.W. et al. Response of Gaucher bone disease to enzyme replacement therapy. Br. J. Radiol. 75, A25, 2002.
-
(2002)
Br. J. Radiol
, vol.75
, pp. A25
-
-
Poll, L.W.1
-
34
-
-
0033843346
-
Children with type I Gaucher disease: Growing into adulthood with and without enzyme therapy
-
Zimran, A., Abrahamov, A., and Elstein, D. Children with type I Gaucher disease: growing into adulthood with and without enzyme therapy. Isr. Med. Assoc. J., 2, 80, 2000.
-
(2000)
Isr. Med. Assoc. J
, vol.2
, pp. 80
-
-
Zimran, A.1
Abrahamov, A.2
Elstein, D.3
-
35
-
-
4444328072
-
Gaucher disease: Alendronate disodium improves bone mineral density in adults receiving enzyme therapy
-
Wenstrup, R.J. et al. Gaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapy. Blood. 104, 1253, 2004.
-
(2004)
Blood
, vol.104
, pp. 1253
-
-
Wenstrup, R.J.1
-
36
-
-
0037159549
-
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry
-
Weinreb, N.J., et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. Am. J. Med., 113, 112, 2002.
-
(2002)
Am. J. Med
, vol.113
, pp. 112
-
-
Weinreb, N.J.1
-
38
-
-
0242468277
-
Routine magnetic resonance imaging of the spine in children with Gaucher disease: Does it help therapeutic management?
-
Vellodi, A, Routine magnetic resonance imaging of the spine in children with Gaucher disease: does it help therapeutic management?, Pediatr. Radiol., 33, 782, 2003.
-
(2003)
Pediatr. Radiol
, vol.33
, pp. 782
-
-
Vellodi, A.1
-
39
-
-
0029910836
-
Bone density in Type 1 Gaucher disease
-
Pastores, G.M. et al. Bone density in Type 1 Gaucher disease. J. Bone Miner. Res., 11, 1801, 1996.
-
(1996)
J. Bone Miner. Res
, vol.11
, pp. 1801
-
-
Pastores, G.M.1
-
40
-
-
0036073124
-
Bone complications in children with Gaucher disease
-
Bembi, B. et al. Bone complications in children with Gaucher disease. Br. J. Radiol., 75, A37, 2002.
-
(2002)
Br. J. Radiol
, vol.75
, pp. A37
-
-
Bembi, B.1
-
41
-
-
8744297087
-
Bone density changes with enzyme therapy for Gaucher disease
-
Lebel, U. et al. Bone density changes with enzyme therapy for Gaucher disease. J. Bone Miner. Metab., 22, 597, 2004.
-
(2004)
J. Bone Miner. Metab
, vol.22
, pp. 597
-
-
Lebel, U.1
-
42
-
-
27144540405
-
A comprehensive assessment of renal function in patients with Gaucher disease
-
Becker-Cohen, R.A. et al. A comprehensive assessment of renal function in patients with Gaucher disease. Am. J. Kidney Dis., 46, 837, 2005.
-
(2005)
Am. J. Kidney Dis
, vol.46
, pp. 837
-
-
Becker-Cohen, R.A.1
-
43
-
-
19044364702
-
Gaucher disease with nephrotic syndrome: Response to enzyme replacement therapy
-
Santoro, D., Rosenbloom, B.E., and Cohen, AH. Gaucher disease with nephrotic syndrome: response to enzyme replacement therapy. Am. J. Kidney Dis., 40, E4, 2002.
-
(2002)
Am. J. Kidney Dis
, vol.40
, pp. E4
-
-
Santoro, D.1
Rosenbloom, B.E.2
Cohen, A.H.3
-
44
-
-
0031464244
-
Plasma and metabolic abnormalities in Gaucher’s disease
-
Aerts, J.M. and Hollak, C.E. Plasma and metabolic abnormalities in Gaucher’s disease. Baillieres Clin. Haematol., 10, 691, 1997.
-
(1997)
Baillieres Clin. Haematol
, vol.10
, pp. 691
-
-
Aerts, J.M.1
Hollak, C.E.2
-
45
-
-
0031018508
-
Pro-inflammatory cytokines and the pathogenesis of Gaucher’s disease: Increased release of interleukin-6 and interleukin-10
-
Allen, M.J. et al. Pro-inflammatory cytokines and the pathogenesis of Gaucher’s disease: increased release of interleukin-6 and interleukin-10. QJM. 90, 19, 1997.
-
(1997)
QJM
, vol.90
, pp. 19
-
-
Allen, M.J.1
-
46
-
-
0742306870
-
Effect of enzyme replacement therapy on gammopathies in Gaucher disease
-
Brautbar, A. et al. Effect of enzyme replacement therapy on gammopathies in Gaucher disease. Blood Cells Mol. Dis., 32, 214, 2004.
-
(2004)
Blood Cells Mol. Dis
, vol.32
, pp. 214
-
-
Brautbar, A.1
-
47
-
-
11144354236
-
Rheological determinants in patients with Gaucher disease and internal inflammation
-
Zimran, A. et al. Rheological determinants in patients with Gaucher disease and internal inflammation. Am. J. Hematol., 75, 190, 2004.
-
(2004)
Am. J. Hematol
, vol.75
, pp. 190
-
-
Zimran, A.1
-
48
-
-
0026474627
-
A less costly regimen of alglucerase to treat Gaucher’s disease
-
Figueroa, M.L. et al. A less costly regimen of alglucerase to treat Gaucher’s disease. N. Engl. J. Med., 327, 1632, 1992.
-
(1992)
N. Engl. J. Med
, vol.327
, pp. 1632
-
-
Figueroa, M.L.1
-
49
-
-
0027218354
-
Enzyme therapy in Gaucher disease type 1: Dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months
-
Pastores, G.M., Sibille, A.R., and Grabowski, G.A. Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months. Blood. 82, 408, 1993.
-
(1993)
Blood
, vol.82
, pp. 408
-
-
Pastores, G.M.1
Sibille, A.R.2
Grabowski, G.A.3
-
50
-
-
84883875323
-
Uncertain miracle: A biotech drug extends a life, but at what price?
-
November 16
-
Anand, G, Uncertain miracle: a biotech drug extends a life, but at what price?, The Wall Street Journal, November 16, 2005; Page A1.
-
(2005)
The Wall Street Journal
, pp. A1
-
-
Anand, G.1
-
51
-
-
9144251964
-
Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: Consensus recommendations
-
Charrow, J. et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. J. Pediatr., 144, 114, 2004.
-
(2004)
J. Pediatr
, vol.144
, pp. 114
-
-
Charrow, J.1
-
52
-
-
0033836414
-
Withdrawal of enzyme replacement therapy in Gaucher’s disease
-
Elstein, D. et al. Withdrawal of enzyme replacement therapy in Gaucher’s disease. Br. J. Haematol., 110, 488, 2000.
-
(2000)
Br. J. Haematol
, vol.110
, pp. 488
-
-
Elstein, D.1
-
53
-
-
0000276490
-
Current issues in diagnosis and treatment. NIH Technology Assessment Panel on Gaucher Disease
-
NIH Technology Assessment Panel on Gaucher Disease
-
NIH Technology Assessment Panel on Gaucher Disease. Current issues in diagnosis and treatment. NIH Technology Assessment Panel on Gaucher Disease. JAMA. 27, 548, 1996.
-
(1996)
JAMA
, vol.27
, pp. 548
-
-
|