2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Crigler-Najjar syndrome | non-replicating recombinant adeno-associated viral vector, serotype 8, expressing the 1A1 isoform of the bilirubin-uridine diphosphate glucuronosyltransferase gene | Orphan Designation | - |
| glycogen storage disease II | Adeno-associated viral vector expressing human acid alpha glucosidase gene | Orphan Designation | - |