Treatment of mitochondrial disorders

Abstract

Mitochondria are important intracellular organelles that are widely distributed and have a central role in metabolic activity. Production of adenosine triphosphate by oxidative phosphorylation, is the principal function of mitochondria. Defects of mitochondrial metabolism cause a wide range of human diseases. They may present as single or multisystem disease at any age. Treatment of mitochondrial disorders is a challenge, as there is only symptomatic therapy available. As few randomized and controlled studies are published, much of the experience of the treatment of mitochondrial disease is based upon anecdotal reports and small case series, which demonstrate effect of some of the measures available. In this paper, we review the various treatments studied and practiced in the context of mitochondrial disorders. We will also outline the evidence in favor or against them.

Keywords

Mitochondrial disorder - oxidative phosphorylation - encephalomyopathy