2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Usher syndrome | dual adeno-associated viral vector serotype 8 encoding for human myosin VIIA (MYO7A) | Orphan Designation | - |
| cone-rod dystrophy 3 | dual AAV8.ABCA4 gene replacement therapy for the production of human ABCA4 protein | Orphan Designation | - |
| gallbladder disease 1 | dual AAV8.ABCA4 gene replacement therapy for the production of human ABCA4 protein | Orphan Designation | - |
| retinitis pigmentosa 19 | dual AAV8.ABCA4 gene replacement therapy for the production of human ABCA4 protein | Orphan Designation | - |