New therapeutics for the treatment of glycosphingolipid lysosomal storage diseases.
Glycosphingolipid lysosomal storage diseases are a small but challenging group of human disorders to treat. Although these appear to be monogenic disorders where the catalytic activity of enzymes in glycosphingolipid catabolism is impaired, the presentation and severity of disease is heterogeneous....
| Auteurs principaux: | , , |
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| Format: | Journal article |
| Langue: | English |
| Publié: |
2003
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