An inhibitor of carnitine biosynthesis
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Mildronate is a structural analog of γ-butyrobetaine (γBB), an intermediate in the biosynthesis of carnitine. It blocks carnitine synthesis by inhibiting γBB hydroxylase (IC50 = 62 µM) and, less potently, carnitine acetyltransferase (Ki = 1.6 mM). Through these actions, mildronate reduces the levels of free carnitine and long chain acyl carnitine. This leads to suppressed fatty acid metabolism and mitochondrial uncoupling during oxidative conditions, resulting in cardioprotective and neuroprotective effects. Mildronate also improves cognition and reduces amyloid-β pathology in a mouse model of Alzheimer's disease.
LX1606 Hippurate (Telotristat etiprate)
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