1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 15q11q13 microduplication syndrome | Makena | Orphan Designation | - |
| Khan-Khan-Katsanis syndrome | Makena | Orphan Designation | - |
| Marfan syndrome | Makena | Orphan Designation | - |
| Peters plus syndrome | Makena | Orphan Designation | - |
| arteriovenous malformations of the brain | Makena | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Makena | Orphan Designation | - |