Treatment of Mitochondrial Cytopathies

 

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ABSTRACT

Mitochondrial cytopathies are clinically and biochemically heterogeneous disorders affecting energy production. Because of the diverse symptoms spanning organ systems, the large number of biochemical and genetic defects, and an unpredictable clinical course, there are limited data regarding proven effective therapies. In general, treatments for mitochondrial cytopathies are intended to augment energy production as well as reduce the production of free radicals and other toxic metabolites that further limit the generation of cellular energy. Theoretically, treatment can be aimed at increasing respiratory chain activity by supplementing relative deficiencies of cofactors required for proper functioning. Possible strategies to consider may include dietary management, supplemental vitamins and cofactors, and/or specific medications aimed at a particular symptom.

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