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:
Research in Translation
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
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author Beutler, Ernest
author_facet Beutler, Ernest
author_sort Beutler, Ernest
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description 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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spelling pubmed-5294212004-11-30 Enzyme Replacement in Gaucher Disease Beutler, Ernest PLoS Med Research in Translation 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? Public Library of Science 2004-11 2004-11-30 /pmc/articles/PMC529421/ /pubmed/15578103 http://dx.doi.org/10.1371/journal.pmed.0010021 Text en Copyright: © 2004 Ernest Beutler. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are properly credited.
spellingShingle Research in Translation
Beutler, Ernest
Enzyme Replacement in Gaucher Disease
title Enzyme Replacement in Gaucher Disease
title_full Enzyme Replacement in Gaucher Disease
title_fullStr Enzyme Replacement in Gaucher Disease
title_full_unstemmed Enzyme Replacement in Gaucher Disease
title_short Enzyme Replacement in Gaucher Disease
title_sort enzyme replacement in gaucher disease
topic Research in Translation
url 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
work_keys_str_mv AT beutlerernest enzymereplacementingaucherdisease

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