Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Joseph Muenzer, J. E. Wraith, Michael Beck et al. · Genetics in Medicine · 2006 · 585 citations · Full text

Life Sciences, Biochemistry, Enzymology +10

REVERSAL OF CLINICAL FEATURES OF HURLER'S DISEASE AND BIOCHEMICAL IMPROVEMENT AFTER TREATMENT BY BONE-MARROW TRANSPLANTATION

Jacqueline A. Hobbs · The Lancet · 1981 · 494 citations

Medicine, Hematology, Stem Cell Transplantation +6

Mucopolysaccharidosis I: Management and Treatment Guidelines

Joseph Muenzer, J. E. Wraith, L. Clarke · PEDIATRICS · 2008 · 460 citations

Medicine, Hematology, Pediatrics +10

DOUBLE-BLIND COMPARISON OF CAPTOPRIL ALONE AGAINST FRUSEMIDE PLUS AMILORIDE IN MILD HEART FAILURE

A. Richardson, A. Scriven, P A Poole-Wilson et al. · The Lancet · 1987 · 431 citations

Medicine, Cardiology, Pharmacology +8

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey et al. · Molecular Genetics and Metabolism · 2009 · 379 citations

Medicine, Immunology, Pediatrics +5