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ID: ALA4020817
Journal: J Med Chem
Title: N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease.
Authors: D'Alonzo D, De Fenza M, Porto C, Iacono R, Huebecker M, Cobucci-Ponzano B, Priestman DA, Platt F, Parenti G, Moracci M, Palumbo G, Guaragna A.
Abstract: 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 administered singularly or when coincubated with the recombinant human α-glucosidase. In addition, differently from its d-enantiomer, l-NBDNJ does not act as a glycosidase inhibitor.
CiteXplore: 29112434