P Kaminsky1, N Belmatoug, T Billette de Villemeur
1Service de Medecine J (PK), CHU de Nancy, Hopitaux de Brabois, Vandoeuvre.
Gaucher disease is an inherited sphingolipidosis caused by beta-glucosidase deficiency. Enzyme replacement therapy effectively treats Gaucher disease symptoms, improving blood parameters and organ size.
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