-
1
-
-
63149135596
-
Fabry disease
-
Schiffmann R. Fabry disease. Pharmacol Ther 2009: 122: 65-77.
-
(2009)
Pharmacol Ther
, vol.122
, pp. 65-77
-
-
Schiffmann, R.1
-
2
-
-
0042347929
-
Enzyme replacement therapy in an adolescent with Fabry disease
-
Illsinger S, Luecke T, Langen H et al. Enzyme replacement therapy in an adolescent with Fabry disease. Eur J Pediatr 2003: 162: 522-523.
-
(2003)
Eur J Pediatr
, vol.162
, pp. 522-523
-
-
Illsinger, S.1
Luecke, T.2
Langen, H.3
-
3
-
-
5444252085
-
Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease
-
Mills K, Vellodi A, Morris P et al. Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease. Eur J Pediatr 2004: 163: 595-603.
-
(2004)
Eur J Pediatr
, vol.163
, pp. 595-603
-
-
Mills, K.1
Vellodi, A.2
Morris, P.3
-
4
-
-
70349616542
-
Fabry disease in children and the effects of enzyme replacement treatment
-
Pintos-Morell G, Beck M. Fabry disease in children and the effects of enzyme replacement treatment. Eur J Pediatr 2009: 168: 1355-1363.
-
(2009)
Eur J Pediatr
, vol.168
, pp. 1355-1363
-
-
Pintos-Morell, G.1
Beck, M.2
-
5
-
-
33847198320
-
Enzyme replacement therapy with agalsidase alfa in children with Fabry disease
-
Ramaswami U, Wendt S, Pintos-Morell G et al. Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatr 2007: 96: 122-127.
-
(2007)
Acta Paediatr
, vol.96
, pp. 122-127
-
-
Ramaswami, U.1
Wendt, S.2
Pintos-Morell, G.3
-
6
-
-
84859616858
-
Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey
-
Ramaswami U, Parini R, Pintos-Morell G et al. Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey. Clin Genet 2011:81: 485-490.
-
(2011)
Clin Genet
, vol.81
, pp. 485-490
-
-
Ramaswami, U.1
Parini, R.2
Pintos-Morell, G.3
-
7
-
-
33749067655
-
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease
-
Ries M, Clarke JT, Whybra C et al. Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease. Pediatrics 2006: 118: 924-932.
-
(2006)
Pediatrics
, vol.118
, pp. 924-932
-
-
Ries, M.1
Clarke, J.T.2
Whybra, C.3
-
8
-
-
77950519422
-
Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease
-
Schiffmann R, Martin RA, Reimschisel T et al. Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease. J Pediatr 2010: 156: 832-837.
-
(2010)
J Pediatr
, vol.156
, pp. 832-837
-
-
Schiffmann, R.1
Martin, R.A.2
Reimschisel, T.3
-
9
-
-
1442310662
-
Intravenous enzyme substitution therapy in children with Fabry's disease
-
Tondel C, Laegreid LM, Hirth A et al. Intravenous enzyme substitution therapy in children with Fabry's disease. Tidsskr Nor Laegeforen 2003: 123: 3388-3390.
-
(2003)
Tidsskr Nor Laegeforen
, vol.123
, pp. 3388-3390
-
-
Tondel, C.1
Laegreid, L.M.2
Hirth, A.3
-
10
-
-
40849094670
-
Safety and efficacy of enzyme replacement therapy with agalsidase-beta: an international, open-label study in pediatric patients with Fabry disease
-
Wraith JE, Tylki-Szymanska A, Guffon N et al. Safety and efficacy of enzyme replacement therapy with agalsidase-beta: an international, open-label study in pediatric patients with Fabry disease. J Pediatr 2008: 152: 563-570.
-
(2008)
J Pediatr
, vol.152
, pp. 563-570
-
-
Wraith, J.E.1
Tylki-Szymanska, A.2
Guffon, N.3
-
11
-
-
79952748856
-
Safety of agalsidase-alfa in patients with Fabry disease under 7 years
-
Ramaswami U, Parini R, Kampmann C et al. Safety of agalsidase-alfa in patients with Fabry disease under 7 years. Acta Paediatr 2011: 100: 605-611.
-
(2011)
Acta Paediatr
, vol.100
, pp. 605-611
-
-
Ramaswami, U.1
Parini, R.2
Kampmann, C.3
-
12
-
-
0035079412
-
Guidelines for glomerular filtration rate determination in children
-
Piepsz A, Colarinha P, Gordon I et al. Guidelines for glomerular filtration rate determination in children. Eur J Nucl Med 2001: 28: BP31-BP36.
-
(2001)
Eur J Nucl Med
, vol.28
-
-
Piepsz, A.1
Colarinha, P.2
Gordon, I.3
-
13
-
-
0031800927
-
Quantitative analysis of cerebral vasculopathy in patients with Fabry disease
-
Crutchfield KE, Patronas NJ, Dambrosia JM et al. Quantitative analysis of cerebral vasculopathy in patients with Fabry disease. Neurology 1998: 50: 1746-1749.
-
(1998)
Neurology
, vol.50
, pp. 1746-1749
-
-
Crutchfield, K.E.1
Patronas, N.J.2
Dambrosia, J.M.3
-
14
-
-
0347123263
-
White matter lesions in Fabry disease occur in 'prior' selectively hypometabolic and hyperperfused brain regions
-
Moore DF, Altarescu G, Barker WC et al. White matter lesions in Fabry disease occur in 'prior' selectively hypometabolic and hyperperfused brain regions. Brain Res Bull 2003: 62: 231-240.
-
(2003)
Brain Res Bull
, vol.62
, pp. 231-240
-
-
Moore, D.F.1
Altarescu, G.2
Barker, W.C.3
-
15
-
-
70350509103
-
Enzyme replacement therapy for Fabry disease: a systematic review of available evidence
-
Schaefer RM, Tylki-Szymanska A, Hilz MJ. Enzyme replacement therapy for Fabry disease: a systematic review of available evidence. Drugs 2009: 69: 2179-2205.
-
(2009)
Drugs
, vol.69
, pp. 2179-2205
-
-
Schaefer, R.M.1
Tylki-Szymanska, A.2
Hilz, M.J.3
-
16
-
-
12944265457
-
Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease
-
Schiffmann R, Murray GJ, Treco D et al. Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease. Proc Natl Acad Sci U S A 2000: 97: 365-370.
-
(2000)
Proc Natl Acad Sci U S A
, vol.97
, pp. 365-370
-
-
Schiffmann, R.1
Murray, G.J.2
Treco, D.3
-
17
-
-
69049085440
-
Functional and structural nerve fiber findings in heterozygote patients with Fabry disease
-
Torvin MA, Winther BF, Feldt-Rasmussen U et al. Functional and structural nerve fiber findings in heterozygote patients with Fabry disease. Pain 2009: 145: 237-245.
-
(2009)
Pain
, vol.145
, pp. 237-245
-
-
Torvin, M.A.1
Winther, B.F.2
Feldt-Rasmussen, U.3
-
19
-
-
41149100107
-
Cardiac manifestations of Anderson-Fabry disease in children and adolescents
-
Kampmann C, Wiethoff CM, Whybra C et al. Cardiac manifestations of Anderson-Fabry disease in children and adolescents. Acta Paediatr 2008: 97: 463-469.
-
(2008)
Acta Paediatr
, vol.97
, pp. 463-469
-
-
Kampmann, C.1
Wiethoff, C.M.2
Whybra, C.3
-
20
-
-
43049092306
-
Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria
-
Tondel C, Bostad L, Hirth A et al. Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria. Am J Kidney Dis 2008: 51: 767-776.
-
(2008)
Am J Kidney Dis
, vol.51
, pp. 767-776
-
-
Tondel, C.1
Bostad, L.2
Hirth, A.3
-
21
-
-
0036266877
-
Natural history and treatment of renal involvement in Fabry disease
-
Branton M, Schiffmann R, Kopp JB. Natural history and treatment of renal involvement in Fabry disease. J Am Soc Nephrol 2002: 13 (Suppl. 2): S139-S143.
-
(2002)
J Am Soc Nephrol
, vol.13
, Issue.SUPPL. 2
-
-
Branton, M.1
Schiffmann, R.2
Kopp, J.B.3
-
22
-
-
78650275925
-
Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy
-
van Breemen MJ, Rombach SM, Dekker N et al. Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy. Biochim Biophys Acta 2011: 1812: 70-76.
-
(2011)
Biochim Biophys Acta
, vol.1812
, pp. 70-76
-
-
van Breemen, M.J.1
Rombach, S.M.2
Dekker, N.3
-
24
-
-
34248190164
-
Sustained, long-term renal stabilization after 54months of agalsidase-beta therapy in patients with Fabry disease
-
Germain DP, Waldek S, Banikazemi M et al. Sustained, long-term renal stabilization after 54months of agalsidase-beta therapy in patients with Fabry disease. J Am Soc Nephrol 2007: 18: 1547-1557.
-
(2007)
J Am Soc Nephrol
, vol.18
, pp. 1547-1557
-
-
Germain, D.P.1
Waldek, S.2
Banikazemi, M.3
|