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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?

Detalles Bibliográficos
Autor principal: Beutler, Ernest
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2004
Materias:
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
Descripción
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?