1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| functional neutrophil defect | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |
| giant neutrophil leukocytes | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |
| hypotonia-failure to thrive-microcephaly syndrome | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |
| insensitivity to pain with hyperplastic Myelinopathy | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |
| neutrophil actin dysfunction | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |
| twin to twin transfusion syndrome | Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase | Orphan Designation | - |