1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against MAGE-A4 antigen | Orphan Designation | - |
| Finnish type amyloidosis | Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against MAGE-A4 antigen | Orphan Designation | - |
| lysinuric protein intolerance | Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against MAGE-A4 antigen | Orphan Designation | - |