MITOCHONDRIAL DISEASE AWARENESS WEEK-Mitochondrial Disease Educational Series- Lesson 4: Part B: Treatment

Treatment for Mitochondrial Disease

Unfortunately, at this time, there are no cures for these disorders.  Mitochondrial disease typically is a progressive disease and most children never live to adulthood. Reference 1

Goals of treatment 

The primary goal for treatment is to:

  • alleviate symptoms
  • slow down the progression of the disease

 Effectiveness of treatment

  • varies from patient to patient, depending on the exact disorder and the severity of the disorder
  • as a general rule, those with mild disorders tend to respond to treatment better than those with severe disorders
  • in some circumstances, the treatment can be tailored specifically to the patient, and that treatment is effective,
  •   whereas in other circumstance, the treatment is “emperic”, meaning that the treatment makes sense, but that the benefit of treatment is not obvious or proven to be effective
  • treatment will not reverse the damage already sustained, such as brain malformations

Benefits of Treatment and Effectiveness of Therapies Vary

  • treatment may be beneficial and noted immediately in some disorders
  • benefit of treatment may take a few months to notice
  • benefit of treatment may never be noticed, but the treatment may be effective in delaying or stopping the progression of the disease
  • some patients may not benefit from therapy

 Key Points to Treatment

  • Never forget there is standard treatment for some  symptoms (anticonvulsant medication for epilepsy, physical therapy for motor problems, etc.)
  • Dietary
  • Vitamins and supplements
  • Avoidance of stressful factors
  • Treatment must be tailored by the patient’s  physician to meet that patient’s need. Many of these therapies are totally ineffective in some mitochondrial disorders and would be a waste  of time, money and effort. In some cases, the treatment could be            dangerous.


This  information  has been taken directly from the Mitochondrial disease reference 1.




United Mitochondrial Disease Foundation

8085   Saltsburg Road, Suite 201 | Pittsburgh, PA 15239 | Toll-Free 1-888-317-UMDF |

P 412-793-8077|F 412-793-6477 |



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