1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| immune dysregulation, autoimmunity, and autoinflammation | autologous patients¿ CD4+ T cells that have been subjected to lentivirus mediated gene transfer of healthy FOXP3 | Orphan Designation | - |
| immune dysregulation-inflammatory bowel disease-arthritis-recurrent infections-lymphopenia syndrome | autologous patients¿ CD4+ T cells that have been subjected to lentivirus mediated gene transfer of healthy FOXP3 | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | autologous patients¿ CD4+ T cells that have been subjected to lentivirus mediated gene transfer of healthy FOXP3 | Orphan Designation | - |