Clinical development of agalsidase-beta for the treatment of Fabry disease;Développement clinique de l'agalsidase β pour le traitement de la maladie de Fabry

Medecine/Sciences

Volumn 21, Issue SPEC. ISS. NOV., 2005, Pages 57-61

  Germain, Dominique P ^a  

^a HÔPITAL EUROPÉEN GEORGES POMPIDOU   (France)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; CREATININE; ERYTHROPOIETIN; GLOBOTRIAOSYLCERAMIDE; INTERFERON; PLACEBO;

CLINICAL RESEARCH; CONFERENCE PAPER; DOSE RESPONSE; DRUG EFFICACY; ENZYME ANALYSIS; ENZYME REPLACEMENT; EVALUATION; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GENETIC DISORDER; GLOMERULUS FILTRATION; HUMAN; SEROCONVERSION; STATISTICAL ANALYSIS; TISSUE PRESERVATION;

EID: 29944446663     PISSN: 07670974     EISSN: None     Source Type: Journal    
DOI: 10.1051/medsci/20052111s57     Document Type: Conference Paper

References (11)

1. Brady RO, Tallman JF, Johnson WG, et al. Replacement therapy for inherited enzyme deficiency. Use of purified ceramidetrihexosidase in Fabry's disease. N Engl J Med 1973; 289: 9-14.
2. Desnick RJ, Dean KJ, Grabowski G, et al. Enzyme therapy in Fabry disease: differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes. Proc Natl Acad Sci USA 1979; 76: 5326-30.
3. Ioannou YA, Bishop DF, Desnick RJ. Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion. J Cell Biol 1992; 119: 1137-50.
4. Ioannou YA, Zeidner KM, Gordon RE, Desnick RJ. Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice. Am J Hum Genet 2001; 68: 14-25.
5. 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: 711-22.
6. Eng CM, Guffon N, Wilcox WR, et al. Safety and efficacy of recombinant human a-galactosidase replacement therapy in Fabry's disease. N Engl J Med 2001; 345: 9-16.
7. 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: 1933-46.
8. Thurberg BL, Randolph Byers H, et al. Monitoring the 3-year efficacy of enzyme replacement therapy in Fabry disease by repeated skin biopsies. J Invest Dermatol 2004; 122: 900-8.
9. Wilcox W, Banikazemi M, Guffon N, et al. Long-term safety and efficacity of enzyme replacement therapy Fabry disease. Am J Hum Genet 2004; 75: 65-74.
10. 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 (Baltimore) 2002; 81: 122-38.
11. Kowey PR, Dickson TZ, Zhang Z, et al. Losartan and end-organ protection: lessons from the RENAAL study. Clin Cardiol 2005; 28: 136-42.