Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.
The inherited metabolic disorders of glycosphingolipid (GSL) metabolism are a relatively rare group of diseases that have diverse and often neurodegenerative phenotypes. Typically, a deficiency in catabolic enzyme activity leads to lysosomal storage of GSL substrates and in many diseases, several ot...
Main Authors: | Butters, T, Dwek, R, Platt, F |
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Format: | Journal article |
Language: | English |
Published: |
2005
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