Mucopolysacharidosis VI (Marateaux-Lamy syndrome) is a lysosomal storage disease that is caused by the deficiency of N-acetylgalactosamine-4-sulfatase (aryl sulfase B, ASB).  The deficiency in ASB causes a build-up in the glycosaminoglycan chondroitin sulfate and dermatan sulfate resulting in debilitating multisystemic organ damage.  Pharmacologic chaperone therapy uses small molecules to rescue the mutant lysosomal enzyme and facilitate its delivery to the lysosome.  In this study, derivatives of praziquantel were synthesized and evaluated as potential small molecule pharmacological chaperones of ASB using enzymatic activity assays.  Studies on the effect of the derivatives of praziquantel on the enzymatic activity of ASB will be presented.

Synthesis and evaluation of Praziquantel derivatives as a potential pharmacological chaperone for Mucopolysacharidosis VI (Marateaux-Lamy syndrome)

248th National Meeting (2014)

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