1
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| beta-thalassemia intermedia | Lentiviral vector encoded with a human beta-globin gene plasmid | Orphan Designation | - |
| beta-thalassemia major | Lentiviral vector encoded with a human beta-globin gene plasmid | Orphan Designation | - |