1
Orphan Designations
2
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 15q11q13 microduplication syndrome | Iwilfin | Orphan Designation | - |
| Danon disease | Iwilfin | Orphan Designation | - |
| Marfan syndrome | Iwilfin | Orphan Designation | - |
| arteriovenous malformations of the brain | Iwilfin | Orphan Designation | - |
| neuroblastoma | Iwilfin | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Iwilfin | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Iwilfin | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Iwilfin | Orphan Designation | - |
| pediatric-onset Graves disease | Iwilfin | Orphan Designation | - |