Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice

The metabolic and molecular bases of inherited disease

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

Life Sciences, Genetics, Molecular Genetics +14

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

Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human α-<scp>l</scp>-Iduronidase (Laronidase)

J. E. Wraith, Michael Beck, Roderick Lane et al. · PEDIATRICS · 2007 · 246 citations

Pharmacy, Pharmacokinetics, Pharmacotherapy +9

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 · 219 citations

Life Sciences, Pharmacotherapy, Enzymology +4