1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| severe Canavan disease | autologous mononuclear cells derived from umbilical cord blood | Orphan Designation | - |
| severe achondroplasia-developmental delay-acanthosis nigricans syndrome | autologous mononuclear cells derived from umbilical cord blood | Orphan Designation | - |
| severe intellectual disability-poor language-strabismus-grimacing face-long fingers syndrome | autologous mononuclear cells derived from umbilical cord blood | Orphan Designation | - |