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Related Experiment Videos

Enzyme replacement therapy for Gaucher disease.

E Beutler1, A Kay, A Saven

  • 1Department of Molecular and Experimental Medicine, Research Institute of Scripps Clinic, La Jolla, CA 92037.

Blood
|September 1, 1991
PubMed
Summary
This summary is machine-generated.

Low-dose enzyme replacement therapy using mannose-terminated glucocerebrosidase (Ceredase) showed significant improvements in liver and lung function for Gaucher disease patients. Skeletal manifestations, however, remained unchanged in this study.

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Area of Science:

  • Biochemistry
  • Genetics
  • Enzyme Replacement Therapy

Background:

  • Gaucher disease is a lysosomal storage disorder.
  • Type I Gaucher disease is the most common and least severe form.
  • Enzyme replacement therapy (ERT) is a treatment option for Gaucher disease.