메뉴 건너뛰기




Volumn 69, Issue 7, 2006, Pages 1216-1221

Clinical benefit of enzyme replacement therapy in Fabry disease

Author keywords

Clinical end points; Enzyme replacement therapy; Fabry disease; Left ventricular hypertrophy; Renal function

Indexed keywords

AGALSIDASE BETA; PENTETATE TECHNETIUM TC 99M;

EID: 33645528253     PISSN: 00852538     EISSN: 15231755     Source Type: Journal    
DOI: 10.1038/sj.ki.5000208     Document Type: Article
Times cited : (149)

References (23)
  • 1
    • 0000889058 scopus 로고    scopus 로고
    • Alpha galactosidase deficiency: Fabry disease
    • Scriver CR, Beaudet AL, Sly WS, Valle D (eds). Mac Graw-Hill: New York
    • Desnick RJ, Ioannou YA, Eng CM. Alpha galactosidase deficiency: Fabry disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic and Molecular Bases of Inherited Disease, 8th edn, Vol. 3 Mac Graw-Hill: New York, 2001 pp. 3733-3774.
    • (2001) The Metabolic and Molecular Bases of Inherited Disease, 8th Edn , vol.3 , pp. 3733-3774
    • Desnick, R.J.1    Ioannou, Y.A.2    Eng, C.M.3
  • 2
    • 0036266877 scopus 로고    scopus 로고
    • Natural history and treatment of renal involvement in Fabry disease
    • Branton MH, Schiffmann R, Kopp JB. Natural history and treatment of renal involvement in Fabry disease. J Am Soc Nephrol 2002; 13(Suppl 2): 139-143.
    • (2002) J Am Soc Nephrol , vol.13 , Issue.2 SUPPL. , pp. 139-143
    • Branton, M.H.1    Schiffmann, R.2    Kopp, J.B.3
  • 3
    • 0035097499 scopus 로고    scopus 로고
    • A phase 1/2 clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studies
    • Eng CM, Banikazemi M, Gordon RE et al. A phase 1/2 clinical trial of enzyme replacement in Fabry disease: pharmacokinetic, substrate clearance, and safety studies. Am J Hum Genet 2001; 68: 711-722.
    • (2001) Am J Hum Genet , vol.68 , pp. 711-722
    • Eng, C.M.1    Banikazemi, M.2    Gordon, R.E.3
  • 4
    • 12944265457 scopus 로고    scopus 로고
    • 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 USA 2000; 97: 365-370.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 365-370
    • Schiffmann, R.1    Murray, G.J.2    Treco, D.3
  • 5
    • 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. Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease. N Engl J Med 2001; 345: 9-16.
    • (2001) N Engl J Med , vol.345 , pp. 9-16
    • Eng, C.M.1    Guffon, N.2    Wilcox, W.R.3
  • 6
    • 0036436320 scopus 로고    scopus 로고
    • Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy
    • Thurberg BL, Rennke H, Colvin RB et al. Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney Int 2002; 62: 1933-1946.
    • (2002) Kidney Int , vol.62 , pp. 1933-1946
    • Thurberg, B.L.1    Rennke, H.2    Colvin, R.B.3
  • 7
    • 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. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Hum Genet 2004; 75: 65-74.
    • (2004) Am J Hum Genet , vol.75 , pp. 65-74
    • Wilcox, W.R.1    Banikazemi, M.2    Guffon, N.3
  • 8
    • 0035816007 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry disease: A randomized controlled trial
    • Schiffmann R, Kopp JB, Austin HA et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001; 285: 2743-2749.
    • (2001) JAMA , vol.285 , pp. 2743-2749
    • Schiffmann, R.1    Kopp, J.B.2    Austin, H.A.3
  • 9
    • 0141765881 scopus 로고    scopus 로고
    • Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease
    • Weidemann F, Breunig F, Beer M et al. Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease. Circulation 2003; 108: 1299-1309.
    • (2003) Circulation , vol.108 , pp. 1299-1309
    • Weidemann, F.1    Breunig, F.2    Beer, M.3
  • 10
    • 0014171084 scopus 로고
    • Angiokeratoma corporis diffusum - Fabry's disease
    • Colombi A, Kostyal A, Bracher R et al. Angiokeratoma corporis diffusum - Fabry's disease. Helv Med Acta 1967; 34: 67-83.
    • (1967) Helv Med Acta , vol.34 , pp. 67-83
    • Colombi, A.1    Kostyal, A.2    Bracher, R.3
  • 11
    • 0036122659 scopus 로고    scopus 로고
    • Natural history of Fabry renal disease: Influence of alpha-galactosidase a activity and genetic mutations on clinical course
    • Branton MH, Schiffmann R, Sabnis SG et al. Natural history of Fabry renal disease: influence of alpha-galactosidase A activity and genetic mutations on clinical course. Medicine 2002; 81: 122-138.
    • (2002) Medicine , vol.81 , pp. 122-138
    • Branton, M.H.1    Schiffmann, R.2    Sabnis, S.G.3
  • 12
    • 0036145366 scopus 로고    scopus 로고
    • Patients with Fabry disease on dialysis in the United States
    • Thadani R, Wolf M, West ML et al. Patients with Fabry disease on dialysis in the United States. Kidney Int 2002; 61: 249-259.
    • (2002) Kidney Int , vol.61 , pp. 249-259
    • Thadani, R.1    Wolf, M.2    West, M.L.3
  • 13
    • 0034082529 scopus 로고    scopus 로고
    • Chronic proteinuric nephropathies: Outcomes and response to treatment in a prospective cohort of 352 patients with different patterns of renal injury
    • Ruggenenti P, Perna A, Gherardi G et al. Chronic proteinuric nephropathies: outcomes and response to treatment in a prospective cohort of 352 patients with different patterns of renal injury. Am J Kidney Dis 2000; 35: 1155-1165.
    • (2000) Am J Kidney Dis , vol.35 , pp. 1155-1165
    • Ruggenenti, P.1    Perna, A.2    Gherardi, G.3
  • 14
    • 10644231988 scopus 로고    scopus 로고
    • Fabry disease: Overall effects of agalsidase alfa treatment
    • Beck M, Ricci R, Widmer U et al. Fabry disease: overall effects of agalsidase alfa treatment. Eur J Clin Invest 2004; 34: 838-844.
    • (2004) Eur J Clin Invest , vol.34 , pp. 838-844
    • Beck, M.1    Ricci, R.2    Widmer, U.3
  • 15
    • 0037452544 scopus 로고    scopus 로고
    • Fabry disease, an under-recognized multisystemic disorder: Expert recommendations for diagnosis, management, and enzyme replacement therapy
    • Desnick RJ, Brady R, Barranger J et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003; 138: 338-346.
    • (2003) Ann Intern Med , vol.138 , pp. 338-346
    • Desnick, R.J.1    Brady, R.2    Barranger, J.3
  • 16
    • 0026099642 scopus 로고
    • An atypical variant of Fabry's disease with manifestations confined to the myocardium
    • von Scheidt W, Eng CM, Fitzmaurice TF et al. An atypical variant of Fabry's disease with manifestations confined to the myocardium. N Engl J Med 1991; 324: 395-399.
    • (1991) N Engl J Med , vol.324 , pp. 395-399
    • Von Scheidt, W.1    Eng, C.M.2    Fitzmaurice, T.F.3
  • 17
    • 12444319931 scopus 로고    scopus 로고
    • Fabry disease: Detection of undiagnosed hemodialysis patients and identification of a 'renal variant' phenotype
    • Nakao S, Kodama C, Takenaka T et al. Fabry disease: detection of undiagnosed hemodialysis patients and identification of a 'renal variant' phenotype. Kidney Int 2003; 64: 801-807.
    • (2003) Kidney Int , vol.64 , pp. 801-807
    • Nakao, S.1    Kodama, C.2    Takenaka, T.3
  • 18
    • 0037461097 scopus 로고    scopus 로고
    • Early detection of Fabry cardiomyopathy by tissue Doppler imaging
    • Pieroni M, Chimenti C, Ricci R et al. Early detection of Fabry cardiomyopathy by tissue Doppler imaging. Circulation 2003; 107: 978-984.
    • (2003) Circulation , vol.107 , pp. 978-984
    • Pieroni, M.1    Chimenti, C.2    Ricci, R.3
  • 19
    • 0343618421 scopus 로고    scopus 로고
    • New insights in cardiac structural changes in patients with Fabry's disease
    • Linhart A, Palecek T, Bultas J et al. New insights in cardiac structural changes in patients with Fabry's disease. Am Heart J 2000; 139: 1101-1108.
    • (2000) Am Heart J , vol.139 , pp. 1101-1108
    • Linhart, A.1    Palecek, T.2    Bultas, J.3
  • 20
    • 0037177166 scopus 로고    scopus 로고
    • Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy
    • Sachdev B, Takenaka T, Teraguchi H et al. Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy. Circulation 2002; 105: 1407-1411.
    • (2002) Circulation , vol.105 , pp. 1407-1411
    • Sachdev, B.1    Takenaka, T.2    Teraguchi, H.3
  • 21
    • 0037032275 scopus 로고    scopus 로고
    • Cardiac manifestations of Anderson-Fabry disease in heterozygous females
    • Kampmann C, Baehner F, Whybra C et al. Cardiac manifestations of Anderson-Fabry disease in heterozygous females. J Am Coll Cardiol 2002; 40: 1668-1674.
    • (2002) J Am Coll Cardiol , vol.40 , pp. 1668-1674
    • Kampmann, C.1    Baehner, F.2    Whybra, C.3
  • 22
    • 0037234314 scopus 로고    scopus 로고
    • Enzyme replacement therapy for Fabry disease: Proving the clinical benefit
    • Breunig F, Wanner C. Enzyme replacement therapy for Fabry disease: proving the clinical benefit. Nephrol Dial Transplant 2003; 18: 7-9.
    • (2003) Nephrol Dial Transplant , vol.18 , pp. 7-9
    • Breunig, F.1    Wanner, C.2
  • 23
    • 0037406311 scopus 로고    scopus 로고
    • Fabry disease: Diagnosis and treatment
    • Breunig F, Weidemann F, Beer M et al. Fabry disease: diagnosis and treatment. Kidney Int 2003; 63(Suppl 84): 181-185.
    • (2003) Kidney Int , vol.63 , Issue.84 SUPPL. , pp. 181-185
    • Breunig, F.1    Weidemann, F.2    Beer, M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.