메뉴 건너뛰기




Volumn 3, Issue , 2012, Pages 33-43

Effect of reduced agalsidase beta dosage in fabry patients: The Australian experience

Author keywords

Agalsidase beta; Enzyme replacement therapy; Fabry disease; Fabry disease patient; Mental component summary

Indexed keywords


EID: 84904266782     PISSN: 21928304     EISSN: 21928312     Source Type: Book Series    
DOI: 10.1007/8904_2011_44     Document Type: Chapter
Times cited : (15)

References (41)
  • 4
    • 0036016096 scopus 로고    scopus 로고
    • Agalsidase alfa–a preparation for enzyme replacement therapy in Anderson-Fabry disease
    • Beck M (2002) Agalsidase alfa–a preparation for enzyme replacement therapy in Anderson-Fabry disease. Expert Opin Investig Drugs 11(6):851–858
    • (2002) Expert Opin Investig Drugs , vol.11 , Issue.6 , pp. 851-858
    • Beck, M.1
  • 5
    • 57449085309 scopus 로고    scopus 로고
    • A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease
    • Benichou B, Goyal S, Sung C, Norfleet AM, O'Brien F (2009) A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease. Mol Genet Metab 96(1):4–12
    • (2009) Mol Genet Metab , vol.96 , Issue.1 , pp. 4-12
    • Benichou, B.1    Goyal, S.2    Sung, C.3    Norfleet, A.M.4    O'brien, F.5
  • 6
    • 0036984003 scopus 로고    scopus 로고
    • Pain assessment: The advantages of using pain scales in lysosomal storage diseases
    • Cleeland CS (2002) Pain assessment: the advantages of using pain scales in lysosomal storage diseases. Acta Paediatr Suppl 91(439):43–47
    • (2002) Acta Paediatr Suppl , vol.91 , Issue.439 , pp. 43-47
    • Cleeland, C.S.1
  • 10
    • 59449103189 scopus 로고    scopus 로고
    • Disease-specific health-related quality of life questionnaires for heart failure: A systematic review with meta-analyses
    • Garin O, Ferrer M, Pont A, Rue M, Kotzeva A, Wiklund I, Van Ganse E, Alonso J (2009) Disease-specific health-related quality of life questionnaires for heart failure: a systematic review with meta-analyses. Qual Life Res 18(1):71–85
    • (2009) Qual Life Res , vol.18 , Issue.1 , pp. 71-85
    • Garin, O.1    Ferrer, M.2    Pont, A.3    Rue, M.4    Kotzeva, A.5    Wiklund, I.6    van Ganse, E.7    Alonso, J.8
  • 13
    • 15044357727 scopus 로고    scopus 로고
    • Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: Data from FOS (Fabry Outcome Survey)
    • Hoffmann B, Garcia de Lorenzo A, Mehta A, Beck M, Widmer U, Ricci R (2005) Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey). J Med Genet 42(3):247–252
    • (2005) J Med Genet , vol.42 , Issue.3 , pp. 247-252
    • Hoffmann, B.1    Garcia de Lorenzo, A.2    Mehta, A.3    Beck, M.4    Widmer, U.5    Ricci, R.6
  • 14
    • 33947316741 scopus 로고    scopus 로고
    • Agalsidase Beta: A review of its use in the management of Fabry disease
    • Keating GM, Simpson D (2007) Agalsidase Beta: a review of its use in the management of Fabry disease. Drugs 67(3):435–455
    • (2007) Drugs , vol.67 , Issue.3 , pp. 435-455
    • Keating, G.M.1    Simpson, D.2
  • 16
    • 4644316602 scopus 로고    scopus 로고
    • Enzyme therapy for Fabry disease: Neutralizing antibodies toward agalsidase alpha and beta
    • Linthorst GE, Hollak CE, Donker-Koopman WE, Strijland A, Aerts JM (2004) Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. Kidney Int 66(4):1589–1595
    • (2004) Kidney Int , 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
  • 17
    • 85060699364 scopus 로고    scopus 로고
    • Guidelines for the treatment of Fabry Disease through the Life Saving Drugs Program
    • LSDP (2010) Guidelines for the treatment of Fabry Disease through the Life Saving Drugs Program, August 2010
    • (2010) August
  • 18
    • 70349316414 scopus 로고    scopus 로고
    • Evaluation of low dose, after a standard therapeutic dose, of agalsidase beta during enzyme replacement therapy in patients with Fabry disease
    • Lubanda JC, Anijalg E, Bzduch V, Thurberg BL, Benichou B, Tylki-Szymanska A (2009) Evaluation of low dose, after a standard therapeutic dose, of agalsidase beta during enzyme replacement therapy in patients with Fabry disease. Genet Med 11(4): 256–264
    • (2009) Genet Med , vol.11 , Issue.4 , pp. 256-264
    • Lubanda, J.C.1    Anijalg, E.2    Bzduch, V.3    Thurberg, B.L.4    Benichou, B.5    Tylki-Szymanska, A.6
  • 20
    • 0036240872 scopus 로고    scopus 로고
    • Assessment of health-related quality-of-life in males with Anderson Fabry Disease before therapeutic intervention
    • Miners AH, Holmes A, Sherr L, Jenkinson C, MacDermot KD (2002) Assessment of health-related quality-of-life in males with Anderson Fabry Disease before therapeutic intervention. Qual Life Res 11(2):127–133
    • (2002) Qual Life Res , vol.11 , Issue.2 , pp. 127-133
    • Miners, A.H.1    Holmes, A.2    Sherr, L.3    Jenkinson, C.4    Macdermot, K.D.5
  • 21
    • 34848819423 scopus 로고    scopus 로고
    • Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy
    • Ohashi T, Sakuma M, Kitagawa T, Suzuki K, Ishige N, Eto Y (2007) Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy. Mol Genet Metab 92(3):271–273
    • (2007) Mol Genet Metab , vol.92 , Issue.3 , pp. 271-273
    • Ohashi, T.1    Sakuma, M.2    Kitagawa, T.3    Suzuki, K.4    Ishige, N.5    Eto, Y.6
  • 22
    • 44649172457 scopus 로고    scopus 로고
    • Reduced alpha-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease
    • Ohashi T, Iizuka S, Ida H, Eto Y (2008) Reduced alpha-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease. Mol Genet Metab 94(3):313–318
    • (2008) Mol Genet Metab , vol.94 , Issue.3 , pp. 313-318
    • Ohashi, T.1    Iizuka, S.2    Ida, H.3    Eto, Y.4
  • 23
    • 0035949059 scopus 로고    scopus 로고
    • Enzyme-replacement therapy for Anderson-Fabry disease
    • Pastores GM, Thadhani R (2001) Enzyme-replacement therapy for Anderson-Fabry disease. Lancet 358(9282):601–603
    • (2001) Lancet , vol.358 , Issue.9282 , pp. 601-603
    • Pastores, G.M.1    Thadhani, R.2
  • 24
    • 34547830275 scopus 로고    scopus 로고
    • Safety and pharmacokinetics of agalsidase alfa in patients with Fabry disease and end-stage renal disease
    • Pastores GM, Boyd E, Crandall K, Whelan A, Piersall L, Barnett N (2007) Safety and pharmacokinetics of agalsidase alfa in patients with Fabry disease and end-stage renal disease. Nephrol Dial Transplant 22(7):1920–1925
    • (2007) Nephrol Dial Transplant , vol.22 , Issue.7 , pp. 1920-1925
    • Pastores, G.M.1    Boyd, E.2    Crandall, K.3    Whelan, A.4    Piersall, L.5    Barnett, N.6
  • 28
    • 70350509103 scopus 로고    scopus 로고
    • Enzyme replacement therapy for Fabry disease: A systematic review of available evidence
    • Schaefer RM, Tylki-Szymanska A, Hilz MJ (2009) Enzyme replacement therapy for Fabry disease: a systematic review of available evidence. Drugs 69(16):2179–2205
    • (2009) Drugs , vol.69 , Issue.16 , pp. 2179-2205
    • Schaefer, R.M.1    Tylki-Szymanska, A.2    Hilz, M.J.3
  • 30
    • 31544456336 scopus 로고    scopus 로고
    • 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 (2006) Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. Nephrol Dial Transplant 21(2):345–354
    • (2006) Nephrol Dial Transplant , vol.21 , Issue.2 , pp. 345-354
    • Schiffmann, R.1    Ries, M.2    Timmons, M.3    Flaherty, J.T.4    Brady, R.O.5
  • 31
    • 34248138939 scopus 로고    scopus 로고
    • Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing
    • Schiffmann R, Askari H, Timmons M, Robinson C, Benko W, Brady RO, Ries M (2007) Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing. J Am Soc Nephrol 18(5): 1576–1583
    • (2007) J am Soc Nephrol , vol.18 , Issue.5 , pp. 1576-1583
    • Schiffmann, R.1    Askari, H.2    Timmons, M.3    Robinson, C.4    Benko, W.5    Brady, R.O.6    Ries, M.7
  • 33
    • 44949167697 scopus 로고    scopus 로고
    • Preservation of renal function in a patient with Fabry nephropathy on enzyme replacement therapy
    • Torra R, Algaba F, Ars E, Santin S, Fernandez-Llama P, Ballarin J (2008) Preservation of renal function in a patient with Fabry nephropathy on enzyme replacement therapy. Clin Nephrol 69(6): 445–449
    • (2008) Clin Nephrol , vol.69 , Issue.6 , pp. 445-449
    • Torra, R.1    Algaba, F.2    Ars, E.3    Santin, S.4    Fernandez-Llama, P.5    Ballarin, J.6
  • 38
    • 11144358101 scopus 로고    scopus 로고
    • The Mainz Severity Score Index: A new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy
    • Whybra C, Kampmann C, Krummenauer F, Ries M, Mengel E, Miebach E, Baehner F, Kim K, Bajbouj M, Schwarting A, Gal A, Beck M (2004) The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy. Clin Genet 65(4):299–307
    • (2004) Clin Genet , vol.65 , Issue.4 , pp. 299-307
    • Whybra, C.1    Kampmann, C.2    Krummenauer, F.3    Ries, M.4    Mengel, E.5    Miebach, E.6    Baehner, F.7    Kim, K.8    Bajbouj, M.9    Schwarting, A.10    Gal, A.11    Beck, M.12
  • 39
    • 68049134975 scopus 로고    scopus 로고
    • A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease
    • Whybra C, Miebach E, Mengel E, Gal A, Baron K, Beck M, Kampmann C (2009) A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease. Genet Med 11(6):441–449
    • (2009) Genet Med , vol.11 , Issue.6 , pp. 441-449
    • Whybra, C.1    Miebach, E.2    Mengel, E.3    Gal, A.4    Baron, K.5    Beck, M.6    Kampmann, C.7


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.