Bone Response to Enzyme Replacement in Gaucher's Disease
Launched by NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS) · Dec 9, 2002
Current as of December 03, 2023
The purpose of this study is to examine the response of the skeleton to repeated infusions of macrophage-targeted glucocerebrosidase (CEREDASE (Trademark) ) in splenectomized patients with type I Gaucher's disease. The magnitude and rate of development of the skeletal response will be monitored non-invasively. Theoretically, an enhanced response should occur in patients supplemented with pharmacologic doses of 1, 25-dihydroxyvitamin D3 (calcitriol), since calcium absorption and enzyme delivery to bone marrow macrophages should be increased in this setting. These issues will be addressed in ...
- Splenectomized Gaucher patients.
- Aged 18-45 who have not received enzyme therapy for at least 1 year.
- No patients with other illnesses (pulmonary, liver, kidney, bone, hematologic).
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
All reviews come from applied patients