2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 7q11.23 microduplication syndrome | Adeno-associated viral vector containing the gene for human coagulation factor IX | Orphan Designation | - |
| cardiac anomalies - developmental delay - facial dysmorphism syndrome | Adeno-associated viral vector containing the gene for human coagulation factor IX | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Adeno-associated viral vector containing the gene for human coagulation factor IX | Orphan Designation | - |