title: Mitochondrial myopathies: developments in treatment.
abstract: OBJECTIVE
  Treatment options for mitochondrial myopathies remain limited despite rapid advances in the understanding of the molecular basis of these conditions. Existing therapies continue to be evaluated and novel treatment strategies are starting to appear on the horizon.
  
  
  RESULTS
  Exercise training continues to show promise as a method of improving exercise tolerance and enhancing oxidative capacity. Coenzyme Q10 deficiency appears to be a relatively common finding in mitochondrial disorders and is likely to benefit from exogenous supplementation. Large-scale randomized clinical trials to evaluate these treatment options are now underway and this represents one of the most important developments in recent years. Activation of the peroxisome proliferator-activated receptor/peroxisome proliferator-activated receptor-gamma coactivator-1alpha pathway has been shown to induce mitochondrial biogenesis leading to a delayed onset of myopathy and prolonged lifespan in mouse models. A ketogenic diet has also been found to induce mitochondrial biogenesis in mice with mitochondrial myopathy.
  
  
  CONCLUSIONS
  Therapeutic trials of exercise training and coenzyme Q10 supplementation should continue to be offered to patients with mitochondrial myopathies pending the results of evaluation in randomized clinical trials. Further investigation of peroxisome proliferator-activated receptor/peroxisome proliferator-activated receptor-gamma coactivator-1alpha pathway activation, ketogenic diets and other new strategies is required.
authors.affiliation: Mitochondrial Research Group, Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, UK.
authors.forename: Adam
authors.initials: A
authors.lastname: Hassani
authors.forename: Rita
authors.initials: R
authors.lastname: Horvath
authors.forename: Patrick F
authors.initials: PF
authors.lastname: Chinnery
mesh_terms: D000818:Animals; D055423:Diet, Ketogenic; D019587:Dietary Supplements; D005081:Exercise Therapy; D006801:Humans; D017240:Mitochondrial Myopathies / Q000235:genetics / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D009154:Mutation; D047495:PPAR gamma / Q000378:metabolism; D014451:Ubiquinone / Q000031:analogs & derivatives / Q000627:therapeutic use; D014815:Vitamins / Q000627:therapeutic use
chemical_list: D047495:PPAR gamma; D014815:Vitamins; D014451:Ubiquinone; C024989:coenzyme Q10