Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

Disease Mechanism, Genetic Disorder, Genetics +6

Enzyme-Replacement Therapy in Mucopolysaccharidosis I

Emil Kakkis, Joseph Muenzer, George E. Tiller et al. · New England Journal of Medicine · 2001 · 670 citations · Full text

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 · 540 citations

Enzyme Replacement Therapy, Recombinant Human α-L-iduronidase, Pathology +4

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years

Monica Sifuentes, Robin Winkler Doroshow, Richard H. Hoft et al. · Molecular Genetics and Metabolism · 2006 · 217 citations

Medicine, Pharmacology, Follow-up Study +1

Cessation of enzyme replacement therapy in Gaucher disease

Karen A. Grinzaid, Eleanor Geller, Soheil L. Hanna et al. · Genetics in Medicine · 2002 · 39 citations

Enzyme Replacement Therapy, Glycobiology, Pathology +4