1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber congenital amaurosis | Recombinant adeno-associated virus (serotype 2) (rAAV2) gene transfer agent expressing RPE65 | Orphan Designation | - |
| Leber congenital amaurosis 6 | Recombinant adeno-associated virus (serotype 2) (rAAV2) gene transfer agent expressing RPE65 | Orphan Designation | - |
| Leber congenital amaurosis 8 | Recombinant adeno-associated virus (serotype 2) (rAAV2) gene transfer agent expressing RPE65 | Orphan Designation | - |