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The Design and Clinical Development of Inhibitors of Glycosphingolipid Synthesis: Will Invention Be the Mother of Necessity?
The treatment of glycosphingolipid storage diseases by synthesis inhibition was first proposed 40 years ago as an alternative approach to enzyme replacement therapy. We have pursued this strategy through the rational design of potent and selective inhibitors of glucosylceramide synthase, the first s...
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Clinical and Climatological Association
2013
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| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3715929/ https://ncbi.nlm.nih.gov/pubmed/23874009 |
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