1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Joubert syndrome 7 | Adeno-associated viral vector expressing human retinitis pigmentosa GTPase regulator-interacting protein 1 (AAV.Anc80.hRPGRIP1) | Orphan Designation | - |
| Leber congenital amaurosis 6 | Adeno-associated viral vector expressing human retinitis pigmentosa GTPase regulator-interacting protein 1 (AAV.Anc80.hRPGRIP1) | Orphan Designation | - |
| cone-rod dystrophy 13 | Adeno-associated viral vector expressing human retinitis pigmentosa GTPase regulator-interacting protein 1 (AAV.Anc80.hRPGRIP1) | Orphan Designation | - |