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The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases
Glycosphingolipid (GSL) storage diseases have been the focus of efforts to develop small molecule therapeutics from design, experimental proof of concept studies, and clinical trials. Two primary alternative strategies that have been pursued include pharmacological chaperones and GSL synthase inhibi...
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| Main Authors: | , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
The American Society for Biochemistry and Molecular Biology
2014
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4076080/ https://ncbi.nlm.nih.gov/pubmed/24534703 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1194/jlr.R047167 |
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