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A short survey on selected β-galactosidase inhibitors as potential pharmacological chaperones for GM1 -gangliosidosis and Morquio B associated mutants of human lysosomal β-galactosidase is provided highlighting recent developments in this particular area of lysosomal storage disorders and orphan diseases. © 2021 The Authors. Published by The Chemical Society of Japan & Wiley-VCH GmbH.


Arnold E Stütz, Martin Thonhofer, Patrick Weber, Andreas Wolfsgruber, Tanja M Wrodnigg. Pharmacological Chaperones for β-Galactosidase Related to GM1 -Gangliosidosis and Morquio B: Recent Advances. Chemical record (New York, N.Y.). 2021 Nov;21(11):2980-2989

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PMID: 34816592

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