N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease

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...

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Bibliographic Details
Main Authors:	D'Alonzo, D, De Fenza, M, Porto, C, Iacono, R, Huebecker, M, Cobucci-Ponzano, B, Priestman, D, Platt, F, Parenti, G, Moracci, M, Palumbo, G, Guaragna, A
Format:	Journal article
Published:	American Chemical Society 2017

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