21. Mitochondrial disease Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 94 / Drugs : 75 - (DrugBank : 31) / Drug target genes : 51 - Drug target pathways : 106
| Other names | Choronic progressive external ophthalmolegia;CPEO;Leigh syndrome;Mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episode;MELAS;Myoclonus epilepsy associated with ragged-red fibers;MERRF;Mitochondrial respiratory chain disorders;Pearson syndrome; | ||
|---|---|---|---|
| Disease group | Metabolic diseases | ||
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Domestic patients
Med expenses recipients (FY2023) |
1,671 patients Age distribution
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| Related info (in Japanese) | |||
| Clinical research form [April 1, 2025 ~] (in Japanese) | Overview, diagnostic criteria, etc. (pdf), Notice (pdf), Notice 2 (pdf), Clinical research form (pdf) | ||
| Specific pediatric chronic diseases, Japan (in Japanese) | 8-4-50. Pyruvate dehydrogenase complex deficiency | ||
| 8-4-51. Pyruvate carboxylase deficiency | |||
| 8-4-52. Fumarase deficiency | |||
| 8-4-53. Succinyl-CoA ligase deficiency | |||
| 8-4-54. Mitochondrial respiratory chain disorders | |||
| 8-4-55. Mitochondrial DNA depletion syndrome | |||
| 8-4-56. Diseases due to mitochondrial DNA mutation | |||
| 8-4-57. Diseases due to mitochondrial DNA deletion | |||
| 8-4-58. Other mitochondrial diseases | |||
| Subtypes | - | ||