Pharmacological Chaperones for β‐Galactosidase Related to G M1 ‐Gangliosidosis and Morquio B: Recent Advances

Authors :: Martin Thonhofer
Andreas Wolfsgruber
Tanja M. Wrodnigg
Patrick Weber
Arnold E. Stütz
Source :: The Chemical Record. 21:2980-2989
Publication Year :: 2021
Publisher :: Wiley, 2021.
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.

Subjects :: General Chemical Engineering
GM1 Gangliosidosis
Lysosomal storage disorders
General Chemistry
Biology
Gangliosidosis
medicine.disease
Orphan diseases
Biochemistry
Pharmacological chaperone
Materials Chemistry
Lysosomal storage disease
medicine
medicine.drug

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