Alpha-Glucosidase Chaperones and Inhibitors for Treatment of Pompe Disease and Type 2 Diabetes
Scientists at the NIH have discovered small molecules that can act as chaperones and correct the misfolding of mutated alpha-glucosidase enzyme. Pompe disease is caused by deficiency or dysfunction of alpha-glucosidase. The only FDA-approved treatment of Pompe disease is enzyme replacement, which is expensive and elicits an immune reaction in most patients that limits clinical utility.
NIH scientists also discovered small molecule inhibitors of alpha glucosidase enzyme. Alpha glucosidase converts carbohydrates into monosaccharides. Inhibition of this conversion is useful for type 2 diabetes. Three FDA-approved inhibitors of alpha glucosidase exist but all have low efficacy:side effect ratios.
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Alpha-Glucosidase Chaperones and Inhibitors for Treatment of Pompe Disease and Type 2 DiabetesLinkback: https://tubagbohol.mikeligalig.com/index.php?topic=65301.0
