1
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult pleomorphic rhabdomyosarcoma | Pheburane | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Pheburane | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Pheburane | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Pheburane | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Pheburane | Orphan Designation | - |
| pediatric-onset Graves disease | Pheburane | Orphan Designation | - |
| urea cycle disorder | Pheburane | Orphan Designation | - |