1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Johanson-Blizzard syndrome | autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 (FOXN1) | Orphan Designation | - |
| congenital | autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 (FOXN1) | Orphan Designation | - |
| congenital heart disease | autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 (FOXN1) | Orphan Designation | - |