1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Kearns-Sayre syndrome | 2-(1H-imidazol-1-yl)-N-(2-(2-methoxyethoxy) pyrimidin-5-yl)-6-(trifluoromethyl) pyrimidine-4-carboxamide | Orphan Designation | - |
| mitochondrial myopathy-cerebellar ataxia-pigmentary retinopathy syndrome | 2-(1H-imidazol-1-yl)-N-(2-(2-methoxyethoxy) pyrimidin-5-yl)-6-(trifluoromethyl) pyrimidine-4-carboxamide | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | 2-(1H-imidazol-1-yl)-N-(2-(2-methoxyethoxy) pyrimidin-5-yl)-6-(trifluoromethyl) pyrimidine-4-carboxamide | Orphan Designation | - |