3-hydroxybutyrate arginate

3-hydroxybutyrate arginate is a prodrug that combines arginine and butyrate through an ester or amide covalent association. It is being investigated as a potential treatment for Duchenne muscular dystrophy (DMD). The drug is designed to enhance the nitric oxide (NO) pathway, primarily through its arginine component, and inhibit histone deacetylase (HDAC) activity via its butyrate component. In preclinical studies using mdx mice and human myotubes, it has shown beneficial effects such as improved force and fatigue resistance, reduced necrosis in heart sections, decreased serum creatine kinase levels, and restoration of membrane integrity by increasing utrophin levels and normalizing lipid composition. These effects were observed at significantly lower doses compared to salt formulations of arginine and butyrate.