1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acquired hemophilia B | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |
| hemophilia A | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |
| hemophilia A with vascular abnormality | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |
| hemophilia B | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |
| hemophilia B leyden | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |
| mild hemophilia B | AAV8/TTR-hFVa - adeno associated virus, serotype 8 with a mouse transthyretin promoter/enhancer and encoded transgene for human clotting Factor Va | Orphan Designation | - |