Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease

Long-term studies of a child with Gaucher disease indicated that the response to treatment with macrophage-targeted glucocerebrosidase (glucosylceramidase) is dose dependent, and that the hematologic response precedes the skeletal response.

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Bibliographische Detailangaben
Veröffentlicht in:	The Journal of pediatrics 1992-02, Vol.120 (2), p.277-280
Hauptverfasser:	Barton, Norman W., Brady, Roscoe O., Dambrosla, James M., Doppelt, Samuel H., Hill, Suvimol C., Holder, Carol A., Mankin, Henry J., Murray, Gary J., Zirzow, Gregory C., Parker, Robert I.
Format:	Artikel
Sprache:	eng
Schlagworte:	Abdomen - pathology Biological and medical sciences Bone and Bones - pathology Child Dose-Response Relationship, Drug Errors of metabolism Follow-Up Studies Gaucher Disease - diagnosis Gaucher Disease - drug therapy Gaucher Disease - enzymology Glucosylceramidase - administration & dosage Glucosylceramidase - blood Humans Lipids (lysosomal enzyme disorders, storage diseases) Macrophages - drug effects Magnetic Resonance Imaging Male Medical sciences Metabolic diseases
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Beschreibung
Zusammenfassung:	Long-term studies of a child with Gaucher disease indicated that the response to treatment with macrophage-targeted glucocerebrosidase (glucosylceramidase) is dose dependent, and that the hematologic response precedes the skeletal response.
ISSN:	0022-3476 1097-6833
DOI:	10.1016/S0022-3476(05)80444-5