A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.

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

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Pharmacology, Toxicology and Pharmaceutical Science