1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| COVID-19–associated multisystem inflammatory syndrome in children | rAAV8-hUGT1A1 | Orphan Designation | - |
| Crigler-Najjar syndrome | rAAV8-hUGT1A1 | Orphan Designation | - |
| cardiovascular disorder | rAAV8-hUGT1A1 | Orphan Designation | - |
| multisystem inflammatory syndrome in children and adults | rAAV8-hUGT1A1 | Orphan Designation | - |
| scrub typhus | rAAV8-hUGT1A1 | Orphan Designation | - |
| tungiasis | rAAV8-hUGT1A1 | Orphan Designation | - |