Small Molecule-Based Enzyme Inhibitors in the Treatment of Primary Hyperoxalurias.

ABSTRACT: Primary hyperoxalurias (PHs) are a group of inherited alterations of the hepatic glyoxylate metabolism. PHs classification based on gene mutations parallel a variety of enzymatic defects, and all involve the harmful accumulation of calcium oxalate crystals that produce systemic damage. These geographically widespread rare diseases have a deep impact in the life quality of the patients. Until recently, treatments were limited to palliative measures and kidney/liver transplants in the most severe forms. Efforts made to develop pharmacological treatments succeeded with the biotechnological agent lumasiran, a siRNA product against glycolate oxidase, which has become the first effective therapy to treat PH1. However, small molecule drugs have classically been preferred since they benefit from e

SUBMITTER: Moya-Garzon MD 

PROVIDER: S-EPMC7912158 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature