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Investigation of original multivalent iminosugars as pharmacological chaperones for the treatment of Gaucher disease.

Carbohydr Res; 429: 98-104, 2016 Jun 24.
Artigo em Inglês | MEDLINE | ID: mdl-27063390
Multivalent iminosugars conjugated with a morpholine moiety and/or designed as prodrugs have been prepared and evaluated as new classes of pharmacological chaperones for the treatment of Gaucher disease. This study further confirms the interest of the prodrug concept and shows that the addition of a lysosome-targeting morpholine unit into iminosugar cluster structures has no significant impact on the chaperone activity on Gaucher cells.