2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| MELAS syndrome | idebenone | Orphan Designation | - |
| congenital muscular dystrophy | omigapil | Orphan Designation | - |
| encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy, 1 | idebenone | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 1 | idebenone | Orphan Designation | - |