Design of multivalent pharmacological chaperones against Pompe disease via metal-free ligation

Key Points

• To design multivalent pharmacological chaperones that target and treat Pompe disease effectively.
• Utilized d- and l-deoxynojirimycin as the basis for pharmacological chaperones.
• Focused on the multivalent design to enhance effectiveness.
• Assessed the potential impact on lysosomal function.
• Successfully designed multivalent pharmacological chaperones.
• Demonstrated improved affinity for targeted proteins related to Pompe disease.
• Showed potential in altering disease pathways associated with acid alpha-glucosidase deficiency.