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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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Dettagli Bibliografici
Pubblicato in:PLoS Med
Autore principale: Beutler, Ernest
Natura: Artigo
Lingua:Inglês
Pubblicazione: PLOS 2004
Soggetti:
Accesso online:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC529421/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/15578103/
https://ncbi.nlm.nih.govhttps://doi.org/10.1371/journal.pmed.0010021
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