Substrate reduction therapy in mouse models of the glycosphingolipidoses.
Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. One of these drugs, N-butyldeoxynojirimycin (NB-DNJ), shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB-DNJ may be of therapeutic benefit, at least i...
| Main Authors: | , , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2003
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