DISEASE SCANNER

Global Incurable Diseases Tracker

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Genetic Disorder

Mitochondrial Disease

HIGH SEVERITY

A group of disorders caused by dysfunction of mitochondria, the cell's energy-producing organelles. Can affect any organ system. Extremely heterogeneous presentation. Can be caused by mitochondrial DNA or nuclear DNA mutations.

Global Affected

1.5M

Countries

15

Symptoms

Muscle weakness
Exercise intolerance
Seizures
Developmental delay
Hearing/vision loss
Diabetes
Cardiomyopathy
Stroke-like episodes
Gastrointestinal dysmotility

Treatment Options

Genetic counseling
Supportive care
Symptom management
Enzyme replacement (if applicable)
Physical therapy
Regular monitoring
Multidisciplinary care

Risk Factors

1Family history
2Genetic mutations
3Consanguinity
4Advanced paternal age
5Ethnic predisposition

Diagnostic Methods

  • 1Genetic testing
  • 2Clinical evaluation
  • 3Family history assessment
  • 4Specialized laboratory tests
  • 5Imaging studies
  • 6Biopsy (if applicable)

Prognosis

Extremely variable by specific mutation and tissue involvement. Progressive course with multi-system involvement typical. Some patients have relatively stable course for years. Supportive care for specific manifestations (seizures, diabetes, cardiac disease). Exercise intolerance common. Avoidance of mitochondrial toxins (valproate, certain antibiotics) important. Coenzyme Q10 and other supplements have limited evidence. Genetic counseling for maternal or Mendelian inheritance depending on mutation.

Prevention

  • Genetic counseling
  • Carrier screening
  • Prenatal diagnosis
  • Preimplantation genetic diagnosis
  • Family planning

Research Status

Supportive care and symptom management. Coenzyme Q10, L-carnitine, B-vitamins sometimes helpful. Avoidance of mitochondrial toxins. No curative treatment. Gene therapy and mitochondrial replacement research ongoing.

Sources

  • https://www.ncbi.nlm.nih.gov/books/NBK1116
  • https://medlineplus.gov/genetics
  • https://rarediseases.org

Medical Disclaimer

This information is for educational purposes only. Always consult healthcare professionals for medical advice, diagnosis, and treatment.