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Pharmacological Chaperones for β-Galactosidase Related to GM1-Gangliosidosis and Morquio B: Recent Advances

Chemical Record (2021) 21(11) 2980-2989
DOI: 10.1002/tcr.202100269
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Abstract

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.

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APA

Stütz, A. E., Thonhofer, M., Weber, P., Wolfsgruber, A., & Wrodnigg, T. M. (2021). Pharmacological Chaperones for β-Galactosidase Related to GM1-Gangliosidosis and Morquio B: Recent Advances. Chemical Record, 21(11), 2980–2989. https://doi.org/10.1002/tcr.202100269

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