1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolf-Hirschhorn syndrome | Allogeneic umbilical cord blood (UCB) cell therapy product consisting of two cell fractions (CD34 enriched and CD34 depleted) derived from the same UCB unit | Orphan Designation | - |
| double nail for fifth toe | Allogeneic umbilical cord blood (UCB) cell therapy product consisting of two cell fractions (CD34 enriched and CD34 depleted) derived from the same UCB unit | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Allogeneic umbilical cord blood (UCB) cell therapy product consisting of two cell fractions (CD34 enriched and CD34 depleted) derived from the same UCB unit | Orphan Designation | - |