Rare Diseases Symptoms Automatic Extraction

Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors.

[gm1 gangliosidosis]

N-Alkylation at the ring nitrogen of the D-galactosidase inhibitor 1-deoxygalactonojirimycin with a functionalised C ₆alkyl chain followed by modification with different aromatic substituents provided lipophilic 1-deoxygalactonojirimycin derivatives which exhibit inhibitory properties against β-glycosidases from E. coli and Agrobacterium sp. as well as green coffee bean α-galactosidase. In preliminary studies, these compounds also showed potential as chemical chaperones for GM1-gangliosidosis related β-galactosidase mutants.

Diseases presenting "different aromatic substituents" symptom

  • gm1 gangliosidosis

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