1
Orphan Designations
1
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 15q11q13 microduplication syndrome | Pedmark | Orphan Designation | - |
| Danon disease | Pedmark | Orphan Designation | - |
| Marfan syndrome | Pedmark | Orphan Designation | - |
| arteriovenous malformations of the brain | Pedmark | Orphan Designation | - |
| platinum-induced ototoxicity | Pedmark | Orphan Designation | - |