Preclinical Efficacy and Safety of 1-Deoxygalactonojirimycin in Mice for Fabry Disease

Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

Christine M. Eng, Nathalie Guffon, William R. Wilcox et al. · New England Journal of Medicine · 2001 · 1.5K citations · Full text

Medicine, Clinical Sciences, Pharmacology +14

Enzyme Replacement Therapy in Fabry Disease

Raphael Schiffmann, Jeffrey B. Kopp, Howard A. Austin et al. · JAMA · 2001 · 1.3K citations

Medicine, Pharmacology, Pharmacotherapy +13

Enzymatic Defect in Fabry's Disease

Roscoe O. Brady, Andrew E. Gal, Roy M. Bradley et al. · New England Journal of Medicine · 1967 · 1.1K citations

Life Sciences, Biochemistry, Dermatology +12

Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

Jianqiang Fan, Naoki Asano, Yoshiyuki Suzuki · Nature Medicine · 1999 · 613 citations

Life Sciences, Biochemistry, Molecular Biology +14

α-Galactosidase A deficiency. Fabry disease

R J Desnick · Medical Entomology and Zoology · 2001 · 581 citations

Life Sciences, Biochemistry, Genetics +5