Small-molecule therapeutics for the treatment of glycolipid lysosomal storage disorders.
Glycosphingolipid (GSL) lysosomal storage disorders are a small but challenging group of human diseases to treat. Although these disorders appear to be monogenic in origin, where the catalytic activity of enzymes in GSL catabolism is impaired, the clinical presentation and severity of disease are he...
| Main Authors: | , , , , |
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| Format: | Journal article |
| Sprog: | English |
| Udgivet: |
2003
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