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- W2606572340 abstract "Abstract Gaucher disease is a group of diverse clinical manifestations involving both the central nervous system and extraneural visceral organs, caused by β-glucosidase deficiency, resulting in massive storage of glucosylceramide. GM1-gagliosidosis is a relatively rare lysosomal disease caused by β-galactosidase deficiency. Both can be treated with chaperone therapy. N-Octyl-β-valienamine (NOV) and N-octyl-4-epi-β-valienamine (NOEV), are two highly potent drug candidates for chemical chaperone therapy for Gaucher disease and GM1-gagliosidosis, respectively. In this chapter, the preparation, physicochemical, and biological characteristics of NOV and NOEV are reviewed. Finally, future perspectives and conclusions for these two drugs are analyzed and drawn." @default.
- W2606572340 created "2017-04-28" @default.
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- W2606572340 date "2017-01-01" @default.
- W2606572340 modified "2023-09-26" @default.
- W2606572340 title "N -Octyl-β-Valienamine and N -Octyl-4- epi -β-Valienamine" @default.
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- W2606572340 doi "https://doi.org/10.1016/b978-0-08-100999-4.00006-x" @default.
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