3
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| myasthenia gravis | autologous anti-CD19 CAR T cell immunotherapy | Orphan Designation | - |
| stiff-person syndrome | autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Orphan Designation | - |
| systemic sclerosis | autologous anti-CD19 CAR T cell immunotherapy transduced with lentiviral vector | Orphan Designation | - |