-
1
-
-
33846908304
-
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial
-
Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, et al.Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Annals of Internal Medicine 2007;146(2):77-86.
-
(2007)
Annals of Internal Medicine
, vol.146
, Issue.2
, pp. 77-86
-
-
Banikazemi, M.1
Bultas, J.2
Waldek, S.3
Wilcox, W.R.4
Whitley, C.B.5
McDonald, M.6
-
2
-
-
33746882747
-
Improvement in serial cardiopulmonary exercise testing following enzyme replacement therapy in Fabry disease
-
Bierer G, Balfe D, Wilcox WR, Mosenifar Z. Improvement in serial cardiopulmonary exercise testing following enzyme replacement therapy in Fabry disease. Journal of Inherited Metabolic Disease 2006;29(4):572-9.
-
(2006)
Journal of Inherited Metabolic Disease
, vol.29
, Issue.4
, pp. 572-579
-
-
Bierer, G.1
Balfe, D.2
Wilcox, W.R.3
Mosenifar, Z.4
-
3
-
-
85048463297
-
Improvement in cardiopulmonary exercise testing during enzyme replacement therapy in fabry disease [Abstract]
-
[B65] [Poster: A45], American Thoracic Society International Conference; 2005
-
Bierer G, Wilcox WR, Balfe D, Mosenifar Z. Improvement in cardiopulmonary exercise testing during enzyme replacement therapy in fabry disease [Abstract]. American Thoracic Society International Conference; 2005. 2005:[B65] [Poster: A45].
-
(2005)
-
-
Bierer, G.1
Wilcox, W.R.2
Balfe, D.3
Mosenifar, Z.4
-
4
-
-
84979710117
-
International Fabry Disease Study Group. Enzyme therapy for Fabry disease [abstract]
-
Desnick RJ. International Fabry Disease Study Group. Enzyme therapy for Fabry disease [abstract]. Journal of Inherited Metabolic Disease 2001;24:774.
-
(2001)
Journal of Inherited Metabolic Disease
, vol.24
, pp. 774
-
-
Desnick, R.J.1
-
5
-
-
84979678118
-
Fabry disease enzyme therapy: phase 3 and extension results [abstract]
-
Desnick RJ, International Fabry Study Group. Fabry disease enzyme therapy: phase 3 and extension results [abstract]. Journal of Inherited Metabolic Disease 2001;24(Suppl 1):98.
-
(2001)
Journal of Inherited Metabolic Disease
, vol.24
, pp. 98
-
-
Desnick, R.J.1
-
6
-
-
0035811624
-
Safety and efficacy of recombinant human alpha-galactosidase A - replacement therapy in Fabry's disease
-
Eng CM, Guffon N, Wilcox WR, Germain DP, Lee P, Waldek S, International Collaborative Fabry Disease Study Group, et al.Safety and efficacy of recombinant human alpha-galactosidase A - replacement therapy in Fabry's disease. New England Journal of Medicine 2001;345(1):9-16.
-
(2001)
New England Journal of Medicine
, vol.345
, Issue.1
, pp. 9-16
-
-
Eng, C.M.1
Guffon, N.2
Wilcox, W.R.3
Germain, D.P.4
Lee, P.5
Waldek, S.6
-
7
-
-
84979669827
-
Severe glomerulosclerosis and proteinuria may influence response to therapy in fabry disease [abstract]
-
Lee P, Banikazemi M, Guffon N, Wilcox WR, Waldek S, Germain DP, et al.Severe glomerulosclerosis and proteinuria may influence response to therapy in fabry disease [abstract]. Journal of Inherited Metabolic Disease 2003;26 Suppl 2:158.
-
(2003)
Journal of Inherited Metabolic Disease
, vol.26
, pp. 158
-
-
Lee, P.1
Banikazemi, M.2
Guffon, N.3
Wilcox, W.R.4
Waldek, S.5
Germain, D.P.6
-
8
-
-
85028484430
-
Aenzyme-supplementation therapy for Fabry disease: first possible treatment [abstract]
-
Linthorst GE, Aerts JMFG, Bosman DK, Heymans HSA, Hollack CEM. Aenzyme-supplementation therapy for Fabry disease: first possible treatment [abstract]. The Netherlands Journal of Medicine 2001;58:A22.
-
(2001)
The Netherlands Journal of Medicine
, vol.58
, pp. A22
-
-
Linthorst, G.E.1
Aerts, J.M.F.G.2
Bosman, D.K.3
Heymans, H.S.A.4
Hollack, C.E.M.5
-
9
-
-
2342544939
-
Monitoring the 3-year efficacy of enzyme replacement therapy in fabry disease by repeated skin biopsies
-
Thurberg BL, Byers RH, Granter SR, Phelps RG, Gordon RE, O'Callaghan M. Monitoring the 3-year efficacy of enzyme replacement therapy in fabry disease by repeated skin biopsies. Journal of Investigative Dermatology 2004;122(4):900-8.
-
(2004)
Journal of Investigative Dermatology
, vol.122
, Issue.4
, pp. 900-908
-
-
Thurberg, B.L.1
Byers, R.H.2
Granter, S.R.3
Phelps, R.G.4
Gordon, R.E.5
O'Callaghan, M.6
-
10
-
-
84979651653
-
Fabrazyme clears globotriaosylceramide from the skin of fabry patients [abstract]
-
Thurberg BL, Phelps R, Granter S, Byers R, Gordon RE, O'Callaghan M. Fabrazyme clears globotriaosylceramide from the skin of fabry patients [abstract]. Journal of Inherited Metabolic Disease 2003;26 Suppl 2:174.
-
(2003)
Journal of Inherited Metabolic Disease
, vol.26
, pp. 174
-
-
Thurberg, B.L.1
Phelps, R.2
Granter, S.3
Byers, R.4
Gordon, R.E.5
O'Callaghan, M.6
-
11
-
-
0036436320
-
Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy
-
Thurberg BL, Rennke H, Colvin RB, Dikman S, Gordon RE, Collins AB, et al.Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney International 2002;62(6):1933-46.
-
(2002)
Kidney International
, vol.62
, Issue.6
, pp. 1933-1946
-
-
Thurberg, B.L.1
Rennke, H.2
Colvin, R.B.3
Dikman, S.4
Gordon, R.E.5
Collins, A.B.6
-
12
-
-
4243056748
-
Stabilization of renal function after enzyme replacement therapy in Fabry Disease [abstract]
-
Waldek S, Germain D P, Banikazemi M, Guffon N, Lee P, Linthorst G, et al.Stabilization of renal function after enzyme replacement therapy in Fabry Disease [abstract]. Nephrology Dialysis Transplantation 2003;18 Suppl 4:630.
-
(2003)
Nephrology Dialysis Transplantation
, vol.18
, pp. 630
-
-
Waldek, S.1
Germain, D.P.2
Banikazemi, M.3
Guffon, N.4
Lee, P.5
Linthorst, G.6
-
13
-
-
3142554529
-
Long-term safety and efficacy of enzyme replacement therapy for Fabry disease
-
Wilcox WR, Banikazemi M, Guffon N, Waldek S, Lee P, Linthorst GE, et al.Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. American Journal of Human Genetics 2004;75(1):65-74.
-
(2004)
American Journal of Human Genetics
, vol.75
, Issue.1
, pp. 65-74
-
-
Wilcox, W.R.1
Banikazemi, M.2
Guffon, N.3
Waldek, S.4
Lee, P.5
Linthorst, G.E.6
-
14
-
-
0942298941
-
Hearing loss in Fabry disease: the effect of agalsidase alfa replacement therapy
-
Hajioff D, Enever Y, Quiney R, Zuckerman J, Mackermot K, Mehta A. Hearing loss in Fabry disease: the effect of agalsidase alfa replacement therapy. Journal of Inherited Metabolic Disease 2003;26(8):787-94.
-
(2003)
Journal of Inherited Metabolic Disease
, vol.26
, Issue.8
, pp. 787-794
-
-
Hajioff, D.1
Enever, Y.2
Quiney, R.3
Zuckerman, J.4
Mackermot, K.5
Mehta, A.6
-
15
-
-
1642496923
-
Hearing improvement in patients with Fabry disease treated with agalsidase alfa
-
Hajioff D, Goodwin S, Quiney R, Zuckerman J, MacDermot KD, Mehta A. Hearing improvement in patients with Fabry disease treated with agalsidase alfa. Acta Paediatrica 2003;92(443):28-30.
-
(2003)
Acta Paediatrica
, vol.92
, Issue.443
, pp. 28-30
-
-
Hajioff, D.1
Goodwin, S.2
Quiney, R.3
Zuckerman, J.4
MacDermot, K.D.5
Mehta, A.6
-
16
-
-
85028484481
-
Hearing loss in Fabry's disease: the effect of alpha-galactosidase A replacement therapy [abstract]
-
Hajioff D, Quiney RE, Zuckerman J, McDermott K Mehta A. Hearing loss in Fabry's disease: the effect of alpha-galactosidase A replacement therapy [abstract]. Annals of Neurology 2003;54 Suppl 7:S26.
-
(2003)
Annals of Neurology
, vol.54
, pp. S26
-
-
Hajioff, D.1
Quiney, R.E.2
Zuckerman, J.3
McDermott, K.M.A.4
-
17
-
-
38749130497
-
Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa
-
Hughes DA, Elliott PM, Shah J, Zuckerman J, Coghlan G, Brookes J, et al.Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart 2008;94(2):153-8.
-
(2008)
Heart
, vol.94
, Issue.2
, pp. 153-158
-
-
Hughes, D.A.1
Elliott, P.M.2
Shah, J.3
Zuckerman, J.4
Coghlan, G.5
Brookes, J.6
-
19
-
-
18244397953
-
Elevated cerebral blood flow velocities in Fabry disease with reversal after enzyme replacement
-
Moore DF, Altarescu G, Ling GS, Jeffries N, Frei KP, Weibel T, et al.Elevated cerebral blood flow velocities in Fabry disease with reversal after enzyme replacement. Stroke 2002;33(2):525-31.
-
(2002)
Stroke
, vol.33
, Issue.2
, pp. 525-531
-
-
Moore, D.F.1
Altarescu, G.2
Ling, G.S.3
Jeffries, N.4
Frei, K.P.5
Weibel, T.6
-
20
-
-
0035949721
-
Regional cerebral hyperperfusion and nitric oxide pathway dysregulation in Fabry disease: reversal by enzyme replacement therapy
-
Moore DF, Scott LTC, Gladwin MT, Altarescu G, Kaneski C, Suzuki K, et al.Regional cerebral hyperperfusion and nitric oxide pathway dysregulation in Fabry disease: reversal by enzyme replacement therapy. Circulation 2001;104(13):1506-12.
-
(2001)
Circulation
, vol.104
, Issue.13
, pp. 1506-1512
-
-
Moore, D.F.1
Scott, L.T.C.2
Gladwin, M.T.3
Altarescu, G.4
Kaneski, C.5
Suzuki, K.6
-
21
-
-
33745686659
-
Enzyme replacement therapy and intraepidermal innervation density in Fabry disease
-
Schiffmann R, Hauer P, Freeman B, Ries M, Scott LJ, Polydefkis M, et al.Enzyme replacement therapy and intraepidermal innervation density in Fabry disease. Muscle Nerve 2006;34(1):53-6.
-
(2006)
Muscle Nerve
, vol.34
, Issue.1
, pp. 53-56
-
-
Schiffmann, R.1
Hauer, P.2
Freeman, B.3
Ries, M.4
Scott, L.J.5
Polydefkis, M.6
-
22
-
-
84979660197
-
Clinical effect of enzyme replacement in Fabry disease [abstract]
-
Schiffmann R, Kopp JB, Austin HA, Sabnis S, Moore DF, Weibel T, et al.Clinical effect of enzyme replacement in Fabry disease [abstract]. Journal of Inherited Metabolic Disease 2001;24(7):775.
-
(2001)
Journal of Inherited Metabolic Disease
, vol.24
, Issue.7
, pp. 775
-
-
Schiffmann, R.1
Kopp, J.B.2
Austin, H.A.3
Sabnis, S.4
Moore, D.F.5
Weibel, T.6
-
23
-
-
0035816007
-
Enzyme replacement therapy in Fabry disease: a randomized controlled trial
-
Schiffmann R, Kopp JB, Austin HA, Sabnis S, Moore DF, Weibel T, et al.Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001;285(21):2743-9.
-
(2001)
JAMA
, vol.285
, Issue.21
, pp. 2743-2749
-
-
Schiffmann, R.1
Kopp, J.B.2
Austin, H.A.3
Sabnis, S.4
Moore, D.F.5
Weibel, T.6
-
24
-
-
39549116083
-
Online CONSORT checklist S1 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]
-
Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ, et al.Online CONSORT checklist S1 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]. PLoS One 2007; Vol. 2, issue 7:e598.
-
(2007)
PLoS One
, vol.2
, Issue.7
-
-
Vedder, A.C.1
Linthorst, G.E.2
Houge, G.3
Groener, J.E.4
Ormel, E.E.5
Bouma, B.J.6
-
25
-
-
39549116083
-
Online supporting information to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]
-
Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ, et al.Online supporting information to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]. PLoS One 2007; Vol. 2, issue 7:e598.
-
(2007)
PLoS One
, vol.2
, Issue.7
-
-
Vedder, A.C.1
Linthorst, G.E.2
Houge, G.3
Groener, J.E.4
Ormel, E.E.5
Bouma, B.J.6
-
26
-
-
39549116083
-
Online trial treatment protocol S1 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]
-
Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ, et al.Online trial treatment protocol S1 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]. PLoS One 2007; Vol. 2, issue 7:e598.
-
(2007)
PLoS One
, vol.2
, Issue.7
-
-
Vedder, A.C.1
Linthorst, G.E.2
Houge, G.3
Groener, J.E.4
Ormel, E.E.5
Bouma, B.J.6
-
27
-
-
39549116083
-
Online trial treatment protocol S2 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]
-
Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ, et al.Online trial treatment protocol S2 to 'Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg' [online]. PLoS One 2007; Vol. 2, issue 7:e598.
-
(2007)
PLoS One
, vol.2
, Issue.7
-
-
Vedder, A.C.1
Linthorst, G.E.2
Houge, G.3
Groener, J.E.4
Ormel, E.E.5
Bouma, B.J.6
-
28
-
-
39549116083
-
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg
-
Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ, et al.Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. PLoS One 2007;2(7):e598.
-
(2007)
PLoS One
, vol.2
, Issue.7
-
-
Vedder, A.C.1
Linthorst, G.E.2
Houge, G.3
Groener, J.E.4
Ormel, E.E.5
Bouma, B.J.6
-
29
-
-
29944435479
-
Enzyme replacement therapy in nine patients with Fabry disease
-
Alamartine E. Enzyme replacement therapy in nine patients with Fabry disease. Medical Science 2005;21 Suppl 11:62-5.
-
(2005)
Medical Science
, vol.21
, pp. 62-65
-
-
Alamartine, E.1
-
30
-
-
23044513293
-
Gastrointestinal manifestations of Fabry disease: clinical response to enzyme replacement therapy
-
Banikasemi M, Ullman T, Desnick RJ. Gastrointestinal manifestations of Fabry disease: clinical response to enzyme replacement therapy. Molecular Genetics and Metabolism 2005;85:255-59.
-
(2005)
Molecular Genetics and Metabolism
, vol.85
, pp. 255-259
-
-
Banikasemi, M.1
Ullman, T.2
Desnick, R.J.3
-
31
-
-
10644231988
-
Fabry disease: overall effects of agalsidase alfa treatment
-
Beck M, Ricci R, Widmer U, Dehout F, Garcia AL, Kampmann C, et al.Fabry disease: overall effects of agalsidase alfa treatment. European Journal of Clinical Investigation 2004;34(12):838-44.
-
(2004)
European Journal of Clinical Investigation
, vol.34
, Issue.12
, pp. 838-844
-
-
Beck, M.1
Ricci, R.2
Widmer, U.3
Dehout, F.4
Garcia, A.L.5
Kampmann, C.6
-
32
-
-
33646173913
-
Impact of enzyme replacement therapy on cardiac morphology and function and late enhancement in Fabry's cardiomyopathy
-
Beer M, Weidemann F, Breunig F, Knoll A, Koeppe S, Machann W, et al.Impact of enzyme replacement therapy on cardiac morphology and function and late enhancement in Fabry's cardiomyopathy. American Journal of Cardiology 2006;97(10):1515-8.
-
(2006)
American Journal of Cardiology
, vol.97
, Issue.10
, pp. 1515-1518
-
-
Beer, M.1
Weidemann, F.2
Breunig, F.3
Knoll, A.4
Koeppe, S.5
Machann, W.6
-
33
-
-
33645528253
-
Clinical benefit of enzyme replacement therapy in Fabry disease
-
Breunig F, Weidemann F, Strotmann J, Knoll A, Wanner C. Clinical benefit of enzyme replacement therapy in Fabry disease. Kidney international 2006;69(7):1216-21.
-
(2006)
Kidney international
, vol.69
, Issue.7
, pp. 1216-1221
-
-
Breunig, F.1
Weidemann, F.2
Strotmann, J.3
Knoll, A.4
Wanner, C.5
-
34
-
-
7244251627
-
Raised HDL cholesterol in Fabry disease: response to enzyme replacement therapy
-
Cartwright DJ, Cole AL, Cousins AJ, Lee PJ. Raised HDL cholesterol in Fabry disease: response to enzyme replacement therapy. Journal of Inherited Metabolic Disease 2004;27(6):791-93.
-
(2004)
Journal of Inherited Metabolic Disease
, vol.27
, Issue.6
, pp. 791-793
-
-
Cartwright, D.J.1
Cole, A.L.2
Cousins, A.J.3
Lee, P.J.4
-
35
-
-
33644524081
-
Coronary microvascular dysfunction in male patients with Anderson-Fabry disease and the effect of treatment with alpha galactosidase A
-
Elliott PM, Kindler H, Shah JS, Sachdev B, Rimoldi OE, Thaman R, et al.Coronary microvascular dysfunction in male patients with Anderson-Fabry disease and the effect of treatment with alpha galactosidase A. Heart 2006;92(3):357-60.
-
(2006)
Heart
, vol.92
, Issue.3
, pp. 357-360
-
-
Elliott, P.M.1
Kindler, H.2
Shah, J.S.3
Sachdev, B.4
Rimoldi, O.E.5
Thaman, R.6
-
36
-
-
19944375153
-
Enzyme replacement therapy in Japanese Fabry disease patients: the results of a phase 2 bridging study
-
Eto Y, Ohashi T, Utsunomiya Y, Fujiwara M, Mizuno A, Inui K, et al.Enzyme replacement therapy in Japanese Fabry disease patients: the results of a phase 2 bridging study. Journal of Inherited Metabolic Disease 2005;28(4):575-83.
-
(2005)
Journal of Inherited Metabolic Disease
, vol.28
, Issue.4
, pp. 575-583
-
-
Eto, Y.1
Ohashi, T.2
Utsunomiya, Y.3
Fujiwara, M.4
Mizuno, A.5
Inui, K.6
-
37
-
-
85028483903
-
Safety and tolerability of agalsidase alfa in patients with Fabry disease formerly treated with agalsidase beta
-
Fernhoff P, Goker-Alpan O, Holida M, Nedd K, Barshop BA, Mardach R, et al.Safety and tolerability of agalsidase alfa in patients with Fabry disease formerly treated with agalsidase beta. Journal of Inherited Metabolic Disease 2011;34(Suppl 3):S227.
-
(2011)
Journal of Inherited Metabolic Disease
, vol.34
, pp. S227
-
-
Fernhoff, P.1
Goker-Alpan, O.2
Holida, M.3
Nedd, K.4
Barshop, B.A.5
Mardach, R.6
-
38
-
-
34248190164
-
Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease
-
Germain DP, Waldek S, Banikazemi M, Bushinsky DA, Charrow J, Desnick RJ, et al.Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease. Journal of the American Society of Nephrology 2007;18(5):1547-57.
-
(2007)
Journal of the American Society of Nephrology
, vol.18
, Issue.5
, pp. 1547-1557
-
-
Germain, D.P.1
Waldek, S.2
Banikazemi, M.3
Bushinsky, D.A.4
Charrow, J.5
Desnick, R.J.6
-
39
-
-
84979710252
-
The clinical benefit of Fabrazyme treatment [abstract]
-
Guffon N. The clinical benefit of Fabrazyme treatment [abstract]. Journal of Inherited Metabolic Disease 2002;25 Suppl 1:116.
-
(2002)
Journal of Inherited Metabolic Disease
, vol.25
, pp. 116
-
-
Guffon, N.1
-
40
-
-
2942560790
-
Clinical benefit in Fabry patients given enzyme replacement therapy - a case series
-
Guffon N, Fouilhoux A. Clinical benefit in Fabry patients given enzyme replacement therapy - a case series. Journal of Inherited Metabolic Disease 2004;27(2):221-7.
-
(2004)
Journal of Inherited Metabolic Disease
, vol.27
, Issue.2
, pp. 221-227
-
-
Guffon, N.1
Fouilhoux, A.2
-
41
-
-
33747078162
-
Agalsidase alpha and hearing in Fabry disease: data from the Fabry Outcome Survey
-
Hajioff D, Hegemannn S, Conti G, Beck M, Sunder-Plassmann G, Widmer U, et al.Agalsidase alpha and hearing in Fabry disease: data from the Fabry Outcome Survey. European Journal of Clinical Investigation 2006;36(9):663-7.
-
(2006)
European Journal of Clinical Investigation
, vol.36
, Issue.9
, pp. 663-667
-
-
Hajioff, D.1
Hegemannn, S.2
Conti, G.3
Beck, M.4
Sunder-Plassmann, G.5
Widmer, U.6
-
42
-
-
1842423556
-
Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy
-
Hilz MJ, Brys M, Marthol H, Stemper B, Dütsch M. Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy. Neurology 2004;62(7):1066-72.
-
(2004)
Neurology
, vol.62
, Issue.7
, pp. 1066-1072
-
-
Hilz, M.J.1
Brys, M.2
Marthol, H.3
Stemper, B.4
Dütsch, M.5
-
43
-
-
33748713855
-
Improvement of sympathetic skin responses under enzyme replacement therapy in Fabry disease
-
Jardim LB, Gomes I, Netto CB, Nora DB, Matte US, Pereira F, et al.Improvement of sympathetic skin responses under enzyme replacement therapy in Fabry disease. Journal of Inherited Metabolic Disease 2006;29(5):653-9.
-
(2006)
Journal of Inherited Metabolic Disease
, vol.29
, Issue.5
, pp. 653-659
-
-
Jardim, L.B.1
Gomes, I.2
Netto, C.B.3
Nora, D.B.4
Matte, U.S.5
Pereira, F.6
-
44
-
-
33750244791
-
White matter lesions in Fabry disease before and after enzyme replacement therapy: a 2-year follow-up
-
Jardim LB, Aesse F, Vedolin LM, Pitta-Pinheiro C, Marconato J, Burin MG, et al.White matter lesions in Fabry disease before and after enzyme replacement therapy: a 2-year follow-up. Arquivos de Neuro-Psiquiatria 2006;64(3B):711-7.
-
(2006)
Arquivos de Neuro-Psiquiatria
, vol.64
, Issue.3B
, pp. 711-717
-
-
Jardim, L.B.1
Aesse, F.2
Vedolin, L.M.3
Pitta-Pinheiro, C.4
Marconato, J.5
Burin, M.G.6
-
45
-
-
33645694070
-
The effect os 12-month enzyme replacement therapy on myocardial perfusion in patients with Fabry disease
-
Kalliokoshi RJ, Kantola I, Kalliokoshi KK, Engblom E, Sundell J, Hannujainen JC, et al.The effect os 12-month enzyme replacement therapy on myocardial perfusion in patients with Fabry disease. Journal of Inherited Metabolic Disease 2006;29:112-8.
-
(2006)
Journal of Inherited Metabolic Disease
, vol.29
, pp. 112-118
-
-
Kalliokoshi, R.J.1
Kantola, I.2
Kalliokoshi, K.K.3
Engblom, E.4
Sundell, J.5
Hannujainen, J.C.6
-
46
-
-
0002494004
-
Influence of enzyme replacement therapy (ERT) on Anderson Fabry disease associated hypertrophic infiltrative cardiomyopathy (HIC)
-
Kampmann C, Ries M, Bahner F, Kim KS, Bajbouj M, Beck M. Influence of enzyme replacement therapy (ERT) on Anderson Fabry disease associated hypertrophic infiltrative cardiomyopathy (HIC). European Journal of Pediatrics 2002;161(2):R5.
-
(2002)
European Journal of Pediatrics
, vol.161
, Issue.2
, pp. R5
-
-
Kampmann, C.1
Ries, M.2
Bahner, F.3
Kim, K.S.4
Bajbouj, M.5
Beck, M.6
-
47
-
-
84979670490
-
Safety of enzyme replacement therapy among 20 Japanese patients with classical type of Fabry disease [abstract]
-
Kobayashi M, Ida H, Ohashi T, Eto Y. Safety of enzyme replacement therapy among 20 Japanese patients with classical type of Fabry disease [abstract]. Journal of Inherited Metabolic Disease 2005;28 Suppl 1:166.
-
(2005)
Journal of Inherited Metabolic Disease
, vol.28
, pp. 166
-
-
Kobayashi, M.1
Ida, H.2
Ohashi, T.3
Eto, Y.4
-
48
-
-
4344671895
-
Enzyme replacement therapy administered during hemodialysis in patients with Fabry disease
-
Kosch M, Koch H-G, Oliveira JP, Soares C, Bianco F, Breuning F, et al.Enzyme replacement therapy administered during hemodialysis in patients with Fabry disease. Kidney International 2004;66(3):1279-82.
-
(2004)
Kidney International
, vol.66
, Issue.3
, pp. 1279-1282
-
-
Kosch, M.1
Koch, H.-G.2
Oliveira, J.P.3
Soares, C.4
Bianco, F.5
Breuning, F.6
-
49
-
-
4644316602
-
Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta
-
Linthorst GE, Hollak CE, Donker-Koopman WE, Strijland A, Aerts JM. Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. Kidney International 2004;66(4):1589-95.
-
(2004)
Kidney International
, vol.66
, Issue.4
, pp. 1589-1595
-
-
Linthorst, G.E.1
Hollak, C.E.2
Donker-Koopman, W.E.3
Strijland, A.4
Aerts, J.M.5
-
50
-
-
31544463530
-
Home treatment for Fabry disease: practice guidelines based on 3 years experience in The Netherlands
-
Linthorst Ge, Vedder AC, Ormel EE, Johannes AMFG, Hollak CEM. Home treatment for Fabry disease: practice guidelines based on 3 years experience in The Netherlands. Nephrology, Dialysis, Transplantation 2006;21:355-360.
-
(2006)
Nephrology, Dialysis, Transplantation
, vol.21
, pp. 355-360
-
-
Linthorst, G.1
Vedder, A.C.2
Ormel, E.E.3
Johannes, A.M.F.G.4
Hollak, C.E.M.5
-
51
-
-
1642463470
-
Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study
-
Mignani R, Panichi V, Giudicissi A, Taccola D, Boscaro F, Feletti C, et al.Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study. Kidney International 2004;65(4):1381-85.
-
(2004)
Kidney International
, vol.65
, Issue.4
, pp. 1381-1385
-
-
Mignani, R.1
Panichi, V.2
Giudicissi, A.3
Taccola, D.4
Boscaro, F.5
Feletti, C.6
-
52
-
-
5444252085
-
Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease
-
Mills K, Vellodi A, Morris P, Cooper D, Morris M, Young E, et al.Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease. European Journal of Pediatrics 2004;163:163.
-
(2004)
European Journal of Pediatrics
, vol.163
, pp. 163
-
-
Mills, K.1
Vellodi, A.2
Morris, P.3
Cooper, D.4
Morris, M.5
Young, E.6
-
53
-
-
20544452974
-
Enzyme replacement therapy in Fabry disease patients undergoing dialysis: effects on quality of life and organ involvement
-
Pisani A, Spinelli L, Sabbatini M, Andreucci MV, Procaccini D, Abbaterusso C, et al.Enzyme replacement therapy in Fabry disease patients undergoing dialysis: effects on quality of life and organ involvement. American Journal of Kidney Diseases 2005;46(1):120-7.
-
(2005)
American Journal of Kidney Diseases
, vol.46
, Issue.1
, pp. 120-127
-
-
Pisani, A.1
Spinelli, L.2
Sabbatini, M.3
Andreucci, M.V.4
Procaccini, D.5
Abbaterusso, C.6
-
54
-
-
33847198320
-
Enzyme replacement therapy with agalsidase alfa in children with Fabry disease
-
Ramaswami U, Wendt S, Pintos-Morell G, Parini R, Whybra C, Leon Leal JA, et al.Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatrica 2007;96(1):122-7.
-
(2007)
Acta Paediatrica
, vol.96
, Issue.1
, pp. 122-127
-
-
Ramaswami, U.1
Wendt, S.2
Pintos-Morell, G.3
Parini, R.4
Whybra, C.5
Leon Leal, J.A.6
-
55
-
-
33749067655
-
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease
-
Ries M, Clarke JT, Whybra C, Timmons M, Robinson C, Schlaggar BL, et al.Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease. Pediatrics 2006;118(3):924-32.
-
(2006)
Pediatrics
, vol.118
, Issue.3
, pp. 924-932
-
-
Ries, M.1
Clarke, J.T.2
Whybra, C.3
Timmons, M.4
Robinson, C.5
Schlaggar, B.L.6
-
56
-
-
0344443401
-
Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease
-
Schiffmann R, Floeter MK, Dambrosia JM, Gupta S, Moore DF, Sharabi Y, et al.Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease. Muscle & Nerve 2003;28(6):703-10.
-
(2003)
Muscle & Nerve
, vol.28
, Issue.6
, pp. 703-710
-
-
Schiffmann, R.1
Floeter, M.K.2
Dambrosia, J.M.3
Gupta, S.4
Moore, D.F.5
Sharabi, Y.6
-
57
-
-
31544456336
-
Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting
-
Schiffmann R, Ries M, Timmons M, Flaherty JT, Brady RO. Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. Nephrology, Dialysis, Transplantation 2006;21(2):345-54.
-
(2006)
Nephrology, Dialysis, Transplantation
, vol.21
, Issue.2
, pp. 345-354
-
-
Schiffmann, R.1
Ries, M.2
Timmons, M.3
Flaherty, J.T.4
Brady, R.O.5
-
58
-
-
4344713083
-
Enzyme replacement therapy with agalsidase beta improves cardiac involvement in Fabry's disease
-
Spinelli L, Pisani A, Sabbatini M, Petretta M, Andreucci MV, Procaccini D, et al.Enzyme replacement therapy with agalsidase beta improves cardiac involvement in Fabry's disease. Clinical Genetics 2004;66:158-65.
-
(2004)
Clinical Genetics
, vol.66
, pp. 158-165
-
-
Spinelli, L.1
Pisani, A.2
Sabbatini, M.3
Petretta, M.4
Andreucci, M.V.5
Procaccini, D.6
-
59
-
-
24044501939
-
Enzyme replacement therapy for Fabry disease: morphologic and histochemical changes in the urinary sediments
-
Utsumi K, Mitsuhashi F, Asahi K, Sakurazawa M, Arii K, Komaba Y, et al.Enzyme replacement therapy for Fabry disease: morphologic and histochemical changes in the urinary sediments. Clinica Chimica Acta 2005;360(1-2):103-7.
-
(2005)
Clinica Chimica Acta
, vol.360
, Issue.1-2
, pp. 103-107
-
-
Utsumi, K.1
Mitsuhashi, F.2
Asahi, K.3
Sakurazawa, M.4
Arii, K.5
Komaba, Y.6
-
60
-
-
0141765881
-
Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: a prospective strain rate imaging study
-
Weidemann F, Breunig F, Beer M, Sandstede J, Turschner O, Voelker W, et al.Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: a prospective strain rate imaging study. Circulation 2003;108(11):1299-301.
-
(2003)
Circulation
, vol.108
, Issue.11
, pp. 1299-1301
-
-
Weidemann, F.1
Breunig, F.2
Beer, M.3
Sandstede, J.4
Turschner, O.5
Voelker, W.6
-
61
-
-
85028484065
-
Agalsidase alfa and agalsidase beta have similar effects on outcomes in Fabry disease - results from the Canadian Fabry disease initiative
-
West M, Bichet D, Casey R, Clarked J, Sirrs S, LeMoine K, et al.Agalsidase alfa and agalsidase beta have similar effects on outcomes in Fabry disease - results from the Canadian Fabry disease initiative. Molecular Genetics and Metabolism 2011;102:S3-S47.
-
(2011)
Molecular Genetics and Metabolism
, vol.102
, pp. S3-S47
-
-
West, M.1
Bichet, D.2
Casey, R.3
Clarked, J.4
Sirrs, S.5
LeMoine, K.6
-
62
-
-
42949119819
-
Elevated globotriaosylsphingosine is a hallmark of Fabry disease
-
Aerts JM, Groener JE, Kuiper S, Donker-Koopman WE, Strijland A, Ottenhoff R, et al.Elevated globotriaosylsphingosine is a hallmark of Fabry disease. Proceedings of the National Academy of Sciences of the United States of America 2008;105(8):2812-7.
-
(2008)
Proceedings of the National Academy of Sciences of the United States of America
, vol.105
, Issue.8
, pp. 2812-2817
-
-
Aerts, J.M.1
Groener, J.E.2
Kuiper, S.3
Donker-Koopman, W.E.4
Strijland, A.5
Ottenhoff, R.6
-
63
-
-
0036211922
-
Meta-analyses involving cross-over trials: methodological issues
-
Elbourne DR, Altman DG, Higgins JPT, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31(1):140-9.
-
(2002)
International Journal of Epidemiology
, vol.31
, Issue.1
, pp. 140-149
-
-
Elbourne, D.R.1
Altman, D.G.2
Higgins, J.P.T.3
Curtin, F.4
Worthington, H.V.5
Vail, A.6
-
64
-
-
0041876133
-
Measuring inconsistency in meta-analyses
-
Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analyses. BMJ 2003;327(7414):557-60.
-
(2003)
BMJ
, vol.327
, Issue.7414
, pp. 557-560
-
-
Higgins, J.P.T.1
Thompson, S.G.2
Deeks, J.J.3
Altman, D.G.4
-
65
-
-
63849105841
-
Cochrane Handbook for Systematic Reviews of Interventions Version 5.0.1 (updated March 2011)
-
The Cochrane Collaboration, 20011
-
Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.0.1 (updated March 2011). The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org 20011.
-
(2011)
-
-
Higgins, J.P.T.1
Green, S.2
-
66
-
-
57649139284
-
Immune response to enzyme replacement therapy in Fabry disease: impact on clinical outcome?
-
Hollak CE, Linthorst GE. Immune response to enzyme replacement therapy in Fabry disease: impact on clinical outcome?. Molecular Genetics and Metabolism 2009;96(1):1-3.
-
(2009)
Molecular Genetics and Metabolism
, vol.96
, Issue.1
, pp. 1-3
-
-
Hollak, C.E.1
Linthorst, G.E.2
-
67
-
-
62149129273
-
Meta-analysis of individual patient data versus aggregate data from longitudinal clinical trials
-
Jones AP. Meta-analysis of individual patient data versus aggregate data from longitudinal clinical trials. Clinical trials 2009;6:16-27.
-
(2009)
Clinical trials
, vol.6
, pp. 16-27
-
-
Jones, A.P.1
-
68
-
-
0033585476
-
Prevalence of lysosomal storage disorders
-
Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA 1999;281(3):249-54.
-
(1999)
JAMA
, vol.281
, Issue.3
, pp. 249-254
-
-
Meikle, P.J.1
Hopwood, J.J.2
Clague, A.E.3
Carey, W.F.4
-
69
-
-
34648833446
-
Enzyme replacement in Fabry disease: pharmacokinetics and pharmacodynamics of agalsidase alpha in children and adolescents
-
Ries M, Clarke JT, Whybra C, Mehta A, Loveday KS, Brady RO, et al.Enzyme replacement in Fabry disease: pharmacokinetics and pharmacodynamics of agalsidase alpha in children and adolescents. Journal of Clinical Pharmacology 2007;47(10):1222-30.
-
(2007)
Journal of Clinical Pharmacology
, vol.47
, Issue.10
, pp. 1222-1230
-
-
Ries, M.1
Clarke, J.T.2
Whybra, C.3
Mehta, A.4
Loveday, K.S.5
Brady, R.O.6
-
70
-
-
0033803473
-
A comparison of four quality of life instruments in cardiac patients: SF-36, QLI, QLMI, and SEIQoL
-
Smith HJ, Taylor R, Mitchell A. A comparison of four quality of life instruments in cardiac patients: SF-36, QLI, QLMI, and SEIQoL. Heart 2000;84(4):390-4.
-
(2000)
Heart
, vol.84
, Issue.4
, pp. 390-394
-
-
Smith, H.J.1
Taylor, R.2
Mitchell, A.3
-
71
-
-
33745280137
-
High incidence of later-onset Fabry disease revealed by newborn screening
-
Spada M, Pagliardini S, Yasuda M, Tukel T, Thiagarajan G, Sakuraba H, et al.High incidence of later-onset Fabry disease revealed by newborn screening. American Journal of Human Genetics 2006;79(1):31-40.
-
(2006)
American Journal of Human Genetics
, vol.79
, Issue.1
, pp. 31-40
-
-
Spada, M.1
Pagliardini, S.2
Yasuda, M.3
Tukel, T.4
Thiagarajan, G.5
Sakuraba, H.6
-
72
-
-
84948769724
-
Investigating and dealing with publication and other biases
-
In: Egger M, Davey Smith G, Altman DG editor(s). London: BMJ Publishing Group
-
Sterne JAC, Egger M, Davey Smith G. Investigating and dealing with publication and other biases. In: Egger M, Davey Smith G, Altman DG editor(s). Systematic Reviews in Health Care. London: BMJ Publishing Group, 2001:189-208.
-
(2001)
Systematic Reviews in Health Care
, pp. 189-208
-
-
Sterne, J.A.C.1
Egger, M.2
Davey Smith, G.3
-
73
-
-
0034192326
-
Misleading funnel plot for detection of bias in meta-analysis
-
Tang JL, Liu JLY. Misleading funnel plot for detection of bias in meta-analysis. Journal of Clinical Epidemiology 2000;53(5):477-84.
-
(2000)
Journal of Clinical Epidemiology
, vol.53
, Issue.5
, pp. 477-484
-
-
Tang, J.L.1
Liu, J.L.Y.2
-
74
-
-
0034112926
-
Publication bias in meta-analysis: its causes and consequences
-
Thornton A, Lee P. Publication bias in meta-analysis: its causes and consequences. Journal of Clinical Epidemiology 2000;53(2):207-16.
-
(2000)
Journal of Clinical Epidemiology
, vol.53
, Issue.2
, pp. 207-216
-
-
Thornton, A.1
Lee, P.2
-
75
-
-
33846265851
-
Heterozygous Fabry women are not just carriers, but have a significant burden of disease and impaired quality of life
-
Wang RY, Lelis A, Mirocha J, Wilcox WR. Heterozygous Fabry women are not just carriers, but have a significant burden of disease and impaired quality of life. Genetics in Medicine 2007;9(1):34-45.
-
(2007)
Genetics in Medicine
, vol.9
, Issue.1
, pp. 34-45
-
-
Wang, R.Y.1
Lelis, A.2
Mirocha, J.3
Wilcox, W.R.4
-
76
-
-
38049036770
-
Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry
-
Wilcox WR, Oliveira JP, Hopkin RJ, Ortiz A, Banikazemi M, Feldt-Rasmussen U, et al.Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry. Molecular Genetics and Metabolism 2008;93(2):112-28.
-
(2008)
Molecular Genetics and Metabolism
, vol.93
, Issue.2
, pp. 112-128
-
-
Wilcox, W.R.1
Oliveira, J.P.2
Hopkin, R.J.3
Ortiz, A.4
Banikazemi, M.5
Feldt-Rasmussen, U.6
-
77
-
-
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, Lien YH, Tsimaratos M, Vellodi A, et al.Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease. Journal of Pediatrics 2008;152(4):563-70.
-
(2008)
Journal of Pediatrics
, vol.152
, Issue.4
, pp. 563-570
-
-
Wraith, J.E.1
Tylki-Szymanska, A.2
Guffon, N.3
Lien, Y.H.4
Tsimaratos, M.5
Vellodi, A.6
|