Therapy for mitochondrial fatty acid beta-oxidation and transport disorders

A method of increasing activity of an enzyme of a β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme, the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme having a missense mutation resulting in loss of its activity or stability, in a patient in need thereof, comprising administering to the patient an effective amount of an inhibitor of activity of an enzyme of the β-oxidation pathway downstream in the β-oxidation pathway of the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme having the mis sense mutation, thereby increasing activity of the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme having the missense mutation, wherein the product, optionally a reverse reaction substrate, of the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme having the missense mutation binds and stabilizes the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme,

• wherein the enzyme of the β-oxidation pathway or fatty acid mitochondrial transporter protein/enzyme is selected from the group consisting of very long chain acyl-CoA dehydrogenase (VLCAD), mitochondrial trifunctional protein (TFP), long-chain 3-ketoacyl-CoA thiolase (LCKAT), and mitochondrial membrane fatty acid transport protein.