1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| hypotonia with lactic acidemia and hyperammonemia | Ucephan | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | Ucephan | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Ucephan | Orphan Designation | - |