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Volumn 9, Issue , 2013, Pages 41-48

Effects of switching from agalsidase beta to agalsidase alfa in 10 patients with anderson-fabry disease

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EID: 84904168429     PISSN: 21928304     EISSN: 21928312     Source Type: Book Series    
DOI: 10.1007/8904_2012_177     Document Type: Chapter
Times cited : (25)

References (41)
  • 1
    • 33846908304 scopus 로고    scopus 로고
    • Agalsidase-beta therapy for advanced Fabry disease: A randomized trial
    • Banikazemi M, Bultas J, Waldek S et al (2007) Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med 146(2):77–86
    • (2007) Ann Intern Med , vol.146 , Issue.2 , pp. 77-86
    • Banikazemi, M.1    Bultas, J.2    Waldek, S.3
  • 2
    • 10644231988 scopus 로고    scopus 로고
    • Fabry disease: Overall effects of agalsidase alfa treatment
    • Beck M, Ricci R, Widmer U et al (2004) Fabry disease: overall effects of agalsidase alfa treatment. Eur J Clin Invest 34(12):838–844
    • (2004) Eur J Clin Invest , vol.34 , Issue.12 , pp. 838-844
    • Beck, M.1    Ricci, R.2    Widmer, U.3
  • 4
    • 0036266877 scopus 로고    scopus 로고
    • Natural history and treatment of renal involvement in Fabry disease
    • Branton M, Schiffmann R, Kopp JB (2002) Natural history and treatment of renal involvement in Fabry disease. J Am Soc Nephrol 13(Suppl 2):S139–143
    • (2002) J am Soc Nephrol , vol.13 , pp. S139-S143
    • Branton, M.1    Schiffmann, R.2    Kopp, J.B.3
  • 6
    • 0000889058 scopus 로고    scopus 로고
    • Alpha-galactosidase A deficiency: Fabry disease
    • Scriver CR, Beaudet AL, Sly WS, Valle D, 8th edn. McGraw-Hill, New York
    • Desnick R, Ioannou Y, Eng C (2001) Alpha-galactosidase A deficiency: Fabry disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The metabolic and molecular bases of inherited disease, 8th edn. McGraw-Hill, New York, pp 3733–3774
    • (2001) The Metabolic and Molecular Bases of Inherited Disease , pp. 3733-3774
    • Desnick, R.1    Ioannou, Y.2    Eng, C.3
  • 7
    • 0015583864 scopus 로고
    • Fabry’s disease: Enzymatic diagnosis of hemizygotes and heterozygotes. Alpha-galactosidase activities in plasma, serum, urine, and leukocytes
    • Desnick RJ, Allen KY, Desnick SJ, Raman MK, Bernlohr RW, Krivit W (1973) Fabry’s disease: enzymatic diagnosis of hemizygotes and heterozygotes. Alpha-galactosidase activities in plasma, serum, urine, and leukocytes. J lLab Clin Med 81(2):157–171
    • (1973) J Llab Clin Med , vol.81 , Issue.2 , pp. 157-171
    • Desnick, R.J.1    Allen, K.Y.2    Desnick, S.J.3    Raman, M.K.4    Bernlohr, R.W.5    Krivit, W.6
  • 8
    • 0035811624 scopus 로고    scopus 로고
    • Safety and efficacy of recombinant human alpha-galactosidase A–replacement therapy in Fabry’s disease
    • Eng CM, Guffon N, Wilcox WR et al (2001) Safety and efficacy of recombinant human alpha-galactosidase A–replacement therapy in Fabry’s disease. N Engl J Med 345(1):9–16
    • (2001) N Engl J Med , vol.345 , Issue.1 , pp. 9-16
    • Eng, C.M.1    Guffon, N.2    Wilcox, W.R.3
  • 9
    • 84988571131 scopus 로고    scopus 로고
    • Accessed 1 June 2012, from
    • European Medicines Agency (2009) Questions and answers on the shortages of Cerezyme and Fabrazyme. Accessed 1 June 2012, from http://www.emea.europa.eu/docs/en_GB/document_library/Medicine_QA/2009/12/WC500018393.pdf
    • (2009) Questions and Answers on the Shortages of Cerezyme and Fabrazyme
  • 10
    • 85060347489 scopus 로고    scopus 로고
    • European Medicines Agency (2010) Assessment report for Fabrazyme agalsidase beta. Accessed 1 June 2012, from www.ema.europa.eu/ docs/en_GB/document_library/Other/2010/11/wc500099241.pdf
    • (2010) Assessment Report for Fabrazyme Agalsidase Beta
  • 11
    • 85014874439 scopus 로고    scopus 로고
    • Accessed 1 June 2012, from
    • European Medicines Agency (2010) European Medicines Agency reviews treatment recommendations for Fabrazyme. Accessed 1 June 2012, from http://www.ema.europa.eu/ema/index.jsp?curl=-pages/news_and_events/news/2010/10/news_detail_001136. jsp&mid=WC0b01ac058004d5c1
    • (2010) European Medicines Agency Reviews Treatment Recommendations for Fabrazyme
  • 12
    • 84857082168 scopus 로고    scopus 로고
    • The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy
    • Feriozzi S, Torras J, Cybulla M et al (2012) The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy. Clin J Am Soc Nephrol 7(1):60–69
    • (2012) Clin J am Soc Nephrol , vol.7 , Issue.1 , pp. 60-69
    • Feriozzi, S.1    Torras, J.2    Cybulla, M.3
  • 13
    • 34248190164 scopus 로고    scopus 로고
    • Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease
    • Germain DP, Waldek S, Banikazemi M et al (2007) Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease. J Am Soc Nephrol 18(5):1547–1557
    • (2007) J am Soc Nephrol , vol.18 , Issue.5 , pp. 1547-1557
    • Germain, D.P.1    Waldek, S.2    Banikazemi, M.3
  • 14
    • 38749130497 scopus 로고    scopus 로고
    • 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 et al (2008) Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart 94(2):153–158
    • (2008) Heart , vol.94 , Issue.2 , pp. 153-158
    • Hughes, D.A.1    Elliott, P.M.2    Shah, J.3
  • 15
    • 67649583702 scopus 로고    scopus 로고
    • Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: A prospective long-term cardiac magnetic resonance imaging study
    • Imbriaco M, Pisani A, Spinelli L et al (2009) Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study. Heart 95(13):1103–1107
    • (2009) Heart , vol.95 , Issue.13 , pp. 1103-1107
    • Imbriaco, M.1    Pisani, A.2    Spinelli, L.3
  • 16
    • 0035163539 scopus 로고    scopus 로고
    • Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice
    • Ioannou YA, Zeidner KM, Gordon RE, Desnick RJ (2001) Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice. Am J Hum Genet 68(1):14–25
    • (2001) Am J Hum Genet , vol.68 , Issue.1 , pp. 14-25
    • Ioannou, Y.A.1    Zeidner, K.M.2    Gordon, R.E.3    Desnick, R.J.4
  • 17
    • 55749096248 scopus 로고    scopus 로고
    • Onset and progression of the Anderson-Fabry disease related cardiomyopathy
    • Kampmann C, Linhart A, Baehner F et al (2008) Onset and progression of the Anderson-Fabry disease related cardiomyopathy. Int J Cardiol 130(3):367–373
    • (2008) Int J Cardiol , vol.130 , Issue.3 , pp. 367-373
    • Kampmann, C.1    Linhart, A.2    Baehner, F.3
  • 18
    • 45849123200 scopus 로고    scopus 로고
    • Twenty-four-month alpha-galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters
    • Koskenvuo JW, Hartiala JJ, Nuutila P et al (2008) Twenty-four-month alpha-galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters. J Inherit Metab Dis 31(3):432–441
    • (2008) J Inherit Metab Dis , vol.31 , Issue.3 , pp. 432-441
    • Koskenvuo, J.W.1    Hartiala, J.J.2    Nuutila, P.3
  • 19
    • 54949143668 scopus 로고    scopus 로고
    • Anderson-Fabry disease: Long-term echocardiographic follow-up under enzyme replacement therapy
    • Kovacevic-Preradovic T, Zuber M, Attenhofer Jost CH et al (2008) Anderson-Fabry disease: long-term echocardiographic follow-up under enzyme replacement therapy. Eur J Echocardiogr 9(6):729–735
    • (2008) Eur J Echocardiogr , vol.9 , Issue.6 , pp. 729-735
    • Kovacevic-Preradovic, T.1    Zuber, M.2    Attenhofer Jost, C.H.3
  • 20
    • 0033574249 scopus 로고    scopus 로고
    • A more accurate method to estimate glomerular filtration rate from serum creatinine: A new prediction equation. Modification of Diet in Renal Disease Study Group
    • Levey AS, Bosch JP, Lewis JB, Greene T, Rogers N, Roth D (1999) A more accurate method to estimate glomerular filtration rate from serum creatinine: a new prediction equation. Modification of Diet in Renal Disease Study Group. Ann Intern Med 130(6):461–470
    • (1999) Ann Intern Med , vol.130 , Issue.6 , pp. 461-470
    • Levey, A.S.1    Bosch, J.P.2    Lewis, J.B.3    Greene, T.4    Rogers, N.5    Roth, D.6
  • 21
    • 78649634506 scopus 로고    scopus 로고
    • Effects of enzyme replacement therapy in Fabry disease–a comprehensive review of the medical literature
    • Lidove O, West ML, Pintos-Morell G et al (2010) Effects of enzyme replacement therapy in Fabry disease–a comprehensive review of the medical literature. Genet Med 12(11):668–679
    • (2010) Genet Med , vol.12 , Issue.11 , pp. 668-679
    • Lidove, O.1    West, M.L.2    Pintos-Morell, G.3
  • 22
    • 38749098726 scopus 로고    scopus 로고
    • Treatment of Anderson-Fabry disease
    • Linhart A (2008) Treatment of Anderson-Fabry disease. Heart 94(2):138–139
    • (2008) Heart , vol.94 , Issue.2 , pp. 138-139
    • Linhart, A.1
  • 23
    • 34247327312 scopus 로고    scopus 로고
    • The heart in Anderson-Fabry disease and other lysosomal storage disorders
    • Linhart A, Elliott PM (2007) The heart in Anderson-Fabry disease and other lysosomal storage disorders. Heart 93(4):528–535
    • (2007) Heart , vol.93 , Issue.4 , pp. 528-535
    • Linhart, A.1    Elliott, P.M.2
  • 24
    • 78650903828 scopus 로고    scopus 로고
    • Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)
    • Linthorst GE, Germain DP, Hollak CE et al (2011) Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT). Mol Genet Metab 102(1):99–102
    • (2011) Mol Genet Metab , vol.102 , Issue.1 , pp. 99-102
    • Linthorst, G.E.1    Germain, D.P.2    Hollak, C.E.3
  • 26
    • 71649111830 scopus 로고    scopus 로고
    • Enzyme replacement therapy with agalsidase alfa in patients with Fabry’s disease: An analysis of registry data
    • Mehta A, Beck M, Elliott P et al (2009) Enzyme replacement therapy with agalsidase alfa in patients with Fabry’s disease: an analysis of registry data. Lancet 374(9706):1986–1996
    • (2009) Lancet , vol.374 , Issue.9706 , pp. 1986-1996
    • Mehta, A.1    Beck, M.2    Elliott, P.3
  • 27
    • 78649632339 scopus 로고    scopus 로고
    • Therapeutic goals in the treatment of Fabry disease
    • Mehta A, West ML, Pintos-Morell G et al (2010) Therapeutic goals in the treatment of Fabry disease. Genet Med 12(11):713–720
    • (2010) Genet Med , vol.12 , Issue.11 , pp. 713-720
    • Mehta, A.1    West, M.L.2    Pintos-Morell, G.3
  • 28
    • 0023576853 scopus 로고
    • The short-form McGill pain questionnaire
    • Melzack R (1987) The short-form McGill pain questionnaire. Pain 30(2):191–197
    • (1987) Pain , vol.30 , Issue.2 , pp. 191-197
    • Melzack, R.1
  • 29
    • 79951595694 scopus 로고    scopus 로고
    • Cardiac abnormalities in Anderson-Fabry disease and Fabry’s cardiomyopathy
    • Morrissey RP, Philip KJ, Schwarz ER (2011) Cardiac abnormalities in Anderson-Fabry disease and Fabry’s cardiomyopathy. Cardiovascular J Africa 22(1):38–44
    • (2011) Cardiovascular J Africa , vol.22 , Issue.1 , pp. 38-44
    • Morrissey, R.P.1    Philip, K.J.2    Schwarz, E.R.3
  • 30
    • 67651111995 scopus 로고    scopus 로고
    • Nephropathy in Fabry disease: The importance of early diagnosis and testing in high-risk populations
    • Oqvist B, Brenner BM, Oliveira JP et al (2009) Nephropathy in Fabry disease: the importance of early diagnosis and testing in high-risk populations. Nephrol Dial Transplant 24(6): 1736–1743
    • (2009) Nephrol Dial Transplant , vol.24 , Issue.6 , pp. 1736-1743
    • Oqvist, B.1    Brenner, B.M.2    Oliveira, J.P.3
  • 32
    • 0035816007 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry disease: A randomized controlled trial
    • Schiffmann R, Kopp JB, Austin HA 3rd et al (2001) Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 285(21):2743–2749
    • (2001) JAMA , vol.285 , Issue.21 , pp. 2743-2749
    • Schiffmann, R.1    Kopp, J.B.2    Austin, H.A.3
  • 33
    • 80054972526 scopus 로고    scopus 로고
    • Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients
    • Smid BE, Rombach SM, Aerts JMFG et al (2011) Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients. Orphanet J Rare Dis 6:69
    • (2011) Orphanet J Rare Dis , vol.6 , pp. 69
    • Smid, B.E.1    Rombach, S.M.2    Aerts, J.M.F.G.3
  • 34
    • 84866106779 scopus 로고    scopus 로고
    • Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagel)
    • Epub ahead of print
    • Tsuboi K, Yamamoto H (2012) Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagel). Genetics Med [Epub ahead of print: doi:101038/gim/2012/39]
    • (2012) Genetics Med
    • Tsuboi, K.1    Yamamoto, H.2
  • 35
    • 44949248124 scopus 로고    scopus 로고
    • Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3
    • Vedder AC, Breunig F, Donker-Koopman WE et al (2008) Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. Mol Genet Metab 94(3): 319–325
    • (2008) Mol Genet Metab , vol.94 , Issue.3 , pp. 319-325
    • Vedder, A.C.1    Breunig, F.2    Donker-Koopman, W.E.3
  • 38
    • 20844452038 scopus 로고    scopus 로고
    • The variation of morphological and functional cardiac manifestation in Fabry disease: Potential implications for the time course of the disease
    • Weidemann F, Breunig F, Beer M et al (2005) The variation of morphological and functional cardiac manifestation in Fabry disease: potential implications for the time course of the disease. Eur Heart J 26(12):1221–1227
    • (2005) Eur Heart J , vol.26 , Issue.12 , pp. 1221-1227
    • Weidemann, F.1    Breunig, F.2    Beer, M.3
  • 39
    • 0141765881 scopus 로고    scopus 로고
    • 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 et al (2003) Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: a prospective strain rate imaging study. Circulation 108(11):1299–1301
    • (2003) Circulation , vol.108 , Issue.11 , pp. 1299-1301
    • Weidemann, F.1    Breunig, F.2    Beer, M.3
  • 40
    • 3142554529 scopus 로고    scopus 로고
    • Long-term safety and efficacy of enzyme replacement therapy for Fabry disease
    • Wilcox WR, Banikazemi M, Guffon N et al (2004) Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Hum Genet 75(1):65–74
    • (2004) Am J Hum Genet , vol.75 , Issue.1 , pp. 65-74
    • Wilcox, W.R.1    Banikazemi, M.2    Guffon, N.3
  • 41
    • 53749104902 scopus 로고    scopus 로고
    • Fabry's disease
    • Zarate YA, Hopkin RJ (2008) Fabry's disease. Lancet 372(9647): 1427–1435
    • (2008) Lancet , vol.372 , Issue.9647 , pp. 1427-1435
    • Zarate, Y.A.1    Hopkin, R.J.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.