1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber congenital amaurosis 2 | rAAV2-CB-hRPE65 | Orphan Designation | - |
| RPE65-related recessive retinopathy | rAAV2-CB-hRPE65 | Orphan Designation | - |
| type II mixed cryoglobulinemia | rAAV2-CB-hRPE65 | Orphan Designation | - |