3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wilson disease | adeno-associated viral vector serotype 3B encoding shortened human ATP7B | Orphan Designation | - |
| inborn disorder of purine or pyrimidine metabolism | adeno-associated viral vector serotype 3B encoding human CYP27A1 | Orphan Designation | - |
| inborn error of immunity | adeno-associated viral vector serotype 3B encoding human CYP27A1 | Orphan Designation | - |
| inborn errors of metabolism | adeno-associated viral vector serotype 3B encoding human CYP27A1 | Orphan Designation | - |
| progressive familial intrahepatic cholestasis | Adeno-associated viral vector encoding human multidrug resistance protein 3A (MDR3A) | Orphan Designation | - |