Enzyme replacement in Gaucher disease.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 15578103.
- Also identified by PMC identifier 529421.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
The development of enzyme replacement therapy for Gaucher disease was a triumph of translational medicine. What were the key steps in its development? What are the controversies surrounding its use?
Medical subject headings
- Gaucher Disease
- Glucosylceramidase