Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.

@article{Weinreb2005GuidanceOT,
  title={Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.},
  author={Neal J Weinreb and John A. Barranger and Joel Charrow and Gregory A. Grabowski and Henry J. Mankin and Pramod K Mistry},
  journal={American journal of hematology},
  year={2005},
  volume={80 3},
  pages={
          223-9
        }
}
Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder due to an autosomal recessive deficiency of glucocerebrosidase. Clinical manifestations include anemia, thrombocytopenia, hepatosplenomegaly, and bone and pulmonary disease. Intravenous enzyme replacement (ERT) with imiglucerase is the accepted standard for treatment of symptomatic patients. More than 3,500 patients worldwide have received ERT with well-documented beneficial effects on the hematological, visceral, skeletal… CONTINUE READING
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