Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C.

Martin Thonhofer, Patrick Weber, Andres Gonzalez Santana, Christina Tysoe, Roland Fischer, Bettina M. Pabst, Eduard Paschke, Michael Schalli, Arnold Stütz, Marion Tschernutter, Werner Windischhofer, Stephen G. Withers

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From an easily available partially protected analog of 1-deoxy-L-gulo-nojirimycin, by chain-branching at C-4 and suitable modification, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have been prepd. New compds. exhibit considerably improved inhibitory activities when compared with the unsubstituted parent compd. and may serve as leads toward new pharmacol. chaperones for GM1-gangliosidosis and Morquio B disease. [on SciFinder(R)]
Original languageEnglish
Pages (from-to)71-80
Number of pages10
JournalCarbohydrate Research
Publication statusPublished - 2016

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