Successful treatment of bone marrow failure in Gaucher's disease with low-dose modified glucocerebrosidase.

@article{Mistry1992SuccessfulTO,
  title={Successful treatment of bone marrow failure in Gaucher's disease with low-dose modified glucocerebrosidase.},
  author={Pramod K Mistry and Sw Davies and Anthony P Corfield and Adrian K. Dixon and T M Cox},
  journal={The Quarterly journal of medicine},
  year={1992},
  volume={83 303},
  pages={
          541-6
        }
}
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. 

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