1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ABCA4-related retinopathy | AAV8_ABCA4 RNA Exon Editor | Orphan Designation | - |
| intrahepatic cholestasis of pregnancy | AAV8_ABCA4 RNA Exon Editor | Orphan Designation | - |
| retinitis pigmentosa 19 | AAV8_ABCA4 RNA Exon Editor | Orphan Designation | - |