Uva-dare (digital Academic Repository) Safety and Efficacy of Recombinant Human Alpha-galactosidase a Replacement Therapy in Fabry's Disease Safety and Efficacy of Recombinant Human a -galactosidase a Replacement Therapy in Fabry's Disease

Disclaimer/Complaints regulations If you believe that digital publication of certain material infringes any of your rights or (privacy) interests, please let the Library know, stating your reasons. In case of a legitimate complaint, the Library will make the material inaccessible and/or remove it from the website. Please Ask the Library: http://uba.uva.nl/en/contact, or a letter to: Library of the University of Amsterdam, Secretariat, Singel 425, 1012 WP Amsterdam, The Netherlands. You will be contacted as soon as possible.

[1]  S. Brodie,et al.  A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies. , 2001, American journal of human genetics.

[2]  G. Bray,et al.  Relationship between Factor VIII Mutation Type and Inhibitor Development in a Cohort of Previously Untreated Patients Treated with Recombinant Factor VIII (Recombinate™) , 2000, Thrombosis and Haemostasis.

[3]  R. Desnick,et al.  Quantitative determination of globotriaosylceramide by immunodetection of glycolipid-bound recombinant verotoxin B subunit. , 1999, Analytical biochemistry.

[4]  N. Leslie,et al.  Enzyme therapy for Gaucher disease: the first 5 years. , 1998, Blood reviews.

[5]  S. Arkin,et al.  Recombinant Factor VIII for the Treatment of Previously Untreated Patients with Hemophilia A: Safety, Efficacy, and Development of Inhibitors , 1993 .

[6]  C. Eng,et al.  An atypical variant of Fabry's disease with manifestations confined to the myocardium. , 1991, The New England journal of medicine.

[7]  M. Elleder,et al.  Cardiocyte storage and hypertrophy as a sole manifestation of Fabry's disease , 1990, Virchows Archiv A.

[8]  Ronald Melzack,et al.  The short-form McGill pain questionnaire , 1987, Pain.

[9]  K. J. Dean,et al.  Enzyme therapy in Fabry disease: differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes. , 1979, Proceedings of the National Academy of Sciences of the United States of America.

[10]  R. Brady,et al.  Replacement therapy for inherited enzyme deficiency. Use of purified ceramidetrihexosidase in Fabry's disease. , 1973, The New England journal of medicine.

[11]  R. Desnick,et al.  Fabry's disease: enzymatic diagnosis of hemizygotes and heterozygotes. Alpha-galactosidase activities in plasma, serum, urine, and leukocytes. , 1973, The Journal of laboratory and clinical medicine.

[12]  R. Desnick,et al.  Diagnosis of glycosphingolipidoses by urinary-sediment analysis. , 1971, The New England journal of medicine.

[13]  R. Desnick,et al.  Enzyme Replacement in Fabry's Disease, an Inborn Error of Metabolism , 1970, Science.

[14]  J. Ware SF-36 health survey: Manual and interpretation guide , 2003 .

[15]  R. Desnick,et al.  Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice. , 2001, American journal of human genetics.

[16]  R. Desnick α-Galactosidase A deficiency. Fabry disease , 2001 .

[17]  P. Mistry Gaucher's disease: a model for modern management of a genetic disease. , 1999, Journal of hepatology.