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Enzyme Replacement in Gaucher Disease
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?
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Formato: | Texto |
Lenguaje: | English |
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Public Library of Science
2004
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC529421/ https://www.ncbi.nlm.nih.gov/pubmed/15578103 http://dx.doi.org/10.1371/journal.pmed.0010021 |
Sumario: | 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? |
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