Q

Genetic Disorders

How do medications treat mitochondrial diseases?

A Answers (2)

  • Currently there are no highly effective mitochondrial disease treatments. Though related symptoms, such as seizures or attention problems can be managed with various medications, the mitochondrial disease itself is unchanged. Unfortunately, supplements such as CoQ10 and L-Carnitine have little to no effect on most patients.

    In 2009, FDA approval occurred for an EID (Emergency Intervention Drug) to treat a mitochondrial disease with the new drug from Edison Pharmaceuticals, EPI-743. This represents the first steps toward viable treatments and hopefully, the beginning of many more near-term advancements in mitochondrial medicine research. 
  • Mitochondrial diseases are treated primarily through vitamin supplements and physical or occupational therapy as opposed to any other kind of medication. Vitamin supplements can include riboflavin, B-complex, coenzyme-Q, carnitine, and vitamin E. These vitamins can help to foster energy levels and help to stave off fatigue in some people with mitochondrial diseases.

This content reflects information from various individuals and organizations and may offer alternative or opposing points of view. It should not be used for medical advice, diagnosis or treatment. As always, you should consult with your healthcare provider about your specific health needs.
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What are the treatment options for mitochondrial diseases?