| Literature DB >> 1484931 |
P K Mistry1, S Davies, A Corfield, A K Dixon, T M Cox.
Abstract
We report the beneficial effects of enzyme replacement therapy with mannose-terminated human glucocerebrosidase ('Ceredase') in a patient suffering from transfusion-dependent bone marrow failure due to Gaucher's disease. Treatment with low-dose enzyme infusions, given twice weekly, rapidly reversed the haematopoietic failure and incapacitating skeletal disease. It appears likely that prior splenectomy favourably influenced the response to this therapy.Entities:
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Year: 1992 PMID： 1484931
Source DB: PubMed Journal: Q J Med ISSN： 0033-5622