NATURAL HISTORY AND TREATMENT OF GAUCHER DISEASE

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a research study to evaluate the natural history and treatment for Gaucher disease. It will seek to determine the safey and efficacy of a treatment regimen in which an initial moderate dose of Ceredase, a commercially available form of placental acid beta-glucosidase is followed by lower maintenance dosing in symptomatic Gaucher disease patients.