1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Achard-Thiers syndrome | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |
| RPGR-related retinopathy | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |
| Wiedemann-Steiner syndrome | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |
| breast cancer | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |
| ovarian cancer | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |
| retinitis pigmentosa 3 | autologous adult bone marrow-derived and unexpanded CD133+ hematopoietic stem cells (CD133+ BMDSCs) mobilized from peripheral blood (PB) | Orphan Designation | - |