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  • Title: [Gaucher disease type 1--therapeutic results of enzyme substitution].
    Author: Steensberg J, Nielsen KG, Brandt NJ.
    Journal: Ugeskr Laeger; 1998 Jun 22; 160(26):3929-30. PubMed ID: 9656836.
    Abstract:
    Gaucher's disease is the most common inherited lysosomal storage disorder, displaying hepato-splenomegaly, thrombocytopenia, anaemia and bone pain as characteristic features. Substitution therapy with a modified enzyme alglucerase has revolutionized the treatment and prognosis of Gaucher's disease. The first Danish patients treated with alglucerase are reported.
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