Article
The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.
Archives of internal medicine - 9 Oct 2000
Charrow J, Andersson H C, Kaplan P, Kolodny E H, Mistry P, Pastores G, Rosenbloom B E, Scott C R, Wappner R S, Weinreb N J, Zimran A
Abstract excerpt
BACKGROUND: The Gaucher Registry, the largest database of patients with Gaucher disease (GD) worldwide, was initiated to better delineate the progressive nature of the disorder and determine optimal therapy. This report describes the demographic and clinical characteristics of 1698 patients with GD before they received enzyme replacement therapy. METHODS: Physicians worldwide who treat patients with GD were...
Topics
- Adolescent
- Adult
- Aged
- Alleles
- Child
- Child, Preschool
- Cross-Cultural Comparison
- Cross-Sectional Studies
- Female
- Gaucher Disease
- Gene Frequency