1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| cone-rod dystrophy 3 | AAV2.GL-3¿ABCA4 + AAV2.GL-5¿ABCA4 | Orphan Designation | - |
| gallbladder disease 1 | AAV2.GL-3¿ABCA4 + AAV2.GL-5¿ABCA4 | Orphan Designation | - |
| retinitis pigmentosa 19 | AAV2.GL-3¿ABCA4 + AAV2.GL-5¿ABCA4 | Orphan Designation | - |