COMPUESTOS POTENCIADORES DE LA ACTIVIDAD DE GLICOSIDASAS MUTANTES

Compounds promoting the activity of mutant glycosidases having inhibitory activity for glycosidase enzymes, furthermore relating to a procedure for activation of mutant ß-glycosidase (ß-glucocerebrosidase) and mutant ß-galactosidase in patients suffering lysosomal storage disorders through administr...

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Hauptverfasser: GARCIA MORENO, MA. ISABEL, GARCIA FERNANDEZ, JOSE MANUEL, OHNO, KOUSAKU, ORTIZ MELLET, CARMEN, AGUILAR MONCAYO, MATILDE, SUZUKI, YOSHIYUKI
Format: Patent
Sprache:spa
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Zusammenfassung:Compounds promoting the activity of mutant glycosidases having inhibitory activity for glycosidase enzymes, furthermore relating to a procedure for activation of mutant ß-glycosidase (ß-glucocerebrosidase) and mutant ß-galactosidase in patients suffering lysosomal storage disorders through administration of said enzyme competitor inhibitor compounds, characterised by very high bonding specificity and a favourable ratio between the concentration thereof for pharmacological chaperone activity and the concentration thereof for inhibitor activity.