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

DOIFull Paper Access

1.3K

Citations

20

References

1991

Year

Norman W. Barton, Roscoe O. Brady, James M. Dambrosia, Adrian M. Di Bisceglie, Samuel H. Doppelt, Suvimol Hill, Henry J. Mankin, Gary J. Murray, Robert I. Parker, Charles E. Argoff,
New England Journal of Medicine

Abstract

Intravenous administration of macrophage-targeted glucocerebrosidase produces objective clinical improvement in patients with type 1 Gaucher's disease. The hematologic and visceral responses to enzyme replacement develop more rapidly than the skeletal response.

References

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