5-(4-(4-Acetylphenyl)piperazin-1-ylsulfonyl)indolin-2-one Analogs as Inhibitors of Acid alpha-Glucosidase for Potential Chaperone Treatment of Pompe Disease or Intervention for Diabetes Mellitus Type 2

Review
In: Probe Reports from the NIH Molecular Libraries Program [Internet]. Bethesda (MD): National Center for Biotechnology Information (US); 2010.
[updated ].

Excerpt

In the field of Lysosomal Storage Disorders, small molecule chaperones are a promising new therapeutic approach that are able to impact the accumulation of functional mutant in the Endoplasmic Reticulum, increasing translocation and ameliorating the disease phenotype. Here, we present the discovery, structure activity relationship (SAR) and initial biological results of a new series of acid alpha glucosidase non-iminosugar inhibitors with chaperone capacity, as exemplified by ML201. In addition, these compounds could potentially be useful for Diabetes type 2 treatment.

Publication types

  • Review