N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease
The highly stereocontrolled de novo synthesis of L-NBDNJ (the unnatural enantiomer of the iminosugar drug Miglustat) and a preliminary evaluation of its chaperoning potential are herein reported. L-NBDNJ is able to enhance lysosomal α-glucosidase levels in Pompe disease fibroblasts, either when admi...
Main Authors: | , , , , , , , , , , , |
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Format: | Journal article |
Published: |
American Chemical Society
2017
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