Related Experiment Videos
[Gaucher disease type 1--therapeutic results of enzyme substitution]
J Steensberg1, K G Nielsen, N J Brandt
1H:S Rigshospitalet, Juliane Marie Centret, afsnit for klinisk genetik.
Ugeskrift for Laeger
|July 10, 1998
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.