Article
The clinical course of untreated Gaucher disease in 22 patients over 10 years: hematological and skeletal manifestations.
Blood cells, molecules & diseases - 1 Jan 2000
Piran Siavash, Roberts Andrew, Patterson Mary Anne, Amato Dominick
Abstract excerpt
Gaucher disease (GD) is a lysosomal storage disorder characterized by anemia and thrombocytopenia, hepatosplenomegaly, and skeletal involvement. The management of Gaucher disease was improved by the development of enzyme replacement therapy (ERT). However, the bone response to ERT is generally slower compared to other clinical manifestations. Some have recommended the early use of ERT to prevent the development...
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