Concepedia

Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice

Kim M. Hemsley, Barbara King, John J. Hopwood

Molecular Genetics and Metabolism · 2006 · 86 citations · 38 references

DOI

Concepts

References

38

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

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Enzyme Replacement Therapy in Fabry Disease

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

, , +13

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

, , +11

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

Kazuhiro Ohmi, David Greenberg, Kavitha S. Rajavel et al. · Proceedings of the National Academy of Sciences · 2003 · 417 citations · Full text

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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

, , +15