2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Stargardt disease 3 | Adeno-associated viral vector serotype 5 containing human ABCA4 gene | Orphan Designation | - |
| Stargardt disease 4 | Adeno-associated viral vector serotype 5 containing human ABCA4 gene | Orphan Designation | - |
| mucopolysaccharidosis type 6 | adeno associated viral vector containing human ARSB gene | Orphan Designation | - |
| mucopolysaccharidosis type 6, rapidly progressing | adeno associated viral vector containing human ARSB gene | Orphan Designation | - |
| mucopolysaccharidosis type 6, slowly progressing | adeno associated viral vector containing human ARSB gene | Orphan Designation | - |
| severe early-childhood-onset retinal dystrophy | Adeno-associated viral vector serotype 5 containing human ABCA4 gene | Orphan Designation | - |