6
Orphan Designations
1
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Omisirge | Orphan Designation | - |
| Hodgkins lymphoma | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Orphan Designation | - |
| acute lymphoblastic leukemia | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Orphan Designation | - |
| acute myeloid leukemia | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Orphan Designation | - |
| cardiomyopathy, familial hypertrophic, 28 | Omisirge | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | NiCord (cord blood-derived exvivo expanded CD34+ cells and the non-expanded cell fraction of the same cord blood unit) | Orphan Designation | - |
| epilepsy, familial adult myoclonic, 1 | Omisirge | Orphan Designation | - |
| epilepsy, familial adult myoclonic, 2 | Omisirge | Orphan Designation | - |
| epilepsy, familial adult myoclonic, 3 | Omisirge | Orphan Designation | - |
| epilepsy, familial adult myoclonic, 4 | Omisirge | Orphan Designation | - |
| immune dysregulation, autoimmunity, and autoinflammation | Omisirge | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | Omisirge | Orphan Designation | - |
| immune reconstitution inflammatory syndrome | Omisirge | Orphan Designation | - |
| immune-mediated scleritis | Omisirge | Orphan Designation | - |
| myelodysplastic syndrome | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Orphan Designation | - |