Concepedia

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

Emil Kakkis, Michael F. McEntee, Carole Vogler, Si-Hung Le, Beth Levy, Pavel V. Belichenko, William C. Mobley, Patricia Dickson, Stephen R. Hanson, Merry Passage

Molecular Genetics and Metabolism · 2004 · 168 citations · 35 references

DOI

Concepts

References

35

The metabolic and molecular bases of inherited disease

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

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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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Convection-enhanced delivery of macromolecules in the brain.

R. Hunt Bobo, Douglas W. Laske, Aytaç Akbaşak et al. · Proceedings of the National Academy of Sciences · 1994 · 1.4K citations

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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

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Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

Norman W. Barton, Roscoe O. Brady, James M. Dambrosia et al. · New England Journal of Medicine · 1991 · 1.3K citations · Full text

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