Mitochondrial Diseases
Diseases caused by abnormal function of the MITOCHONDRIA. They may be caused by mutations, acquired or inherited, in mitochondrial DNA or in nuclear genes that code for mitochondrial components. They may also be the result of acquired mitochondria dysfunction due to adverse effects of drugs, infections, or other environmental causes.
Subtype terms (10)
More specific conditions that are subtypes of Mitochondrial Diseases in the MeSH hierarchy.
- Carbamoyl-Phosphate Synthase I Deficiency Disease
1 drug approved
- Cytochrome-c Oxidase Deficiency
- Friedreich Ataxia
37 drugs (20 approved, 17 experimental)
- Leigh Disease
6 drugs (4 approved, 2 experimental)
- Mitochondrial Myopathies
13 drugs (7 approved, 6 experimental)
- Multiple Acyl Coenzyme A Dehydrogenase Deficiency
2 drugs experimental
- Optic Atrophy, Autosomal Dominant
1 drug experimental
- Optic Atrophy, Hereditary, Leber
19 drugs (3 approved, 16 experimental)
- Pyruvate Carboxylase Deficiency Disease
2 drugs (1 approved, 1 experimental)
- Pyruvate Dehydrogenase Complex Deficiency Disease
4 drugs (3 approved, 1 experimental)
Drugs by status
Approved for this indication (1)
Phase 4 trials (6)
Phase 3 trials (4)
Phase 2 trials (19)
Phase 1 trials (16)
Organizations
Organization Involved with Phase 4 Indications (2)
Organization Involved with Phase 3 Indications (6)
Organization Involved with Phase 2 Indications (14)
- Horizon Pharma
- Bioelectron
- Khondrion BV
- Stealth BioTherapeutics Inc.
- National Human Genome Research Institute (NHGRI)
- University of London
- Newcastle University
- Radboud University
- Stanford University
- Vanderbilt University
- Woodinville Psychiatric Associates
- The Clinical Trial Company
- Neopharm
- Julius Clinical, The Netherlands
Organization Involved with Phase 1 Indications (8)
Organization Involved with Other Experimental Indications (10)