Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease

Enzyme-Replacement Therapy in Mucopolysaccharidosis I

Emil Kakkis, Joseph Muenzer, George E. Tiller et al. · New England Journal of Medicine · 2001 · 675 citations · Full text

Life Sciences, Biochemistry, Genetics +11

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)

J. E. Wraith, L. Clarke, Michael Beck et al. · The Journal of Pediatrics · 2004 · 546 citations

Life Sciences, Biochemistry, Enzymology +7

Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry Disease

William R. Wilcox, Maryam Banikazemi, Nathalie Guffon et al. · The American Journal of Human Genetics · 2004 · 405 citations · Full text

Life Sciences, Pharmacotherapy, Renal Replacement Therapy +7

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease.

Norman W. Barton, F S Furbish, Gary J. Murray et al. · Proceedings of the National Academy of Sciences · 1990 · 370 citations

Medicine, Hematology, Pharmacology +15

Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human α-Glucosidase From Milk

Johanna M. P. van den Hout, Joep H. J. Kamphoven, Léon P. F. Winkel et al. · PEDIATRICS · 2004 · 354 citations

Life Sciences, Biochemistry, Molecular Medicine +12