3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Bio-engineered oral mucosal tissue | Orphan Designation | - |
| anosmia for isobutyric acid | Bio-engineered oral mucosal tissue | Orphan Designation | - |
| conjunctival squamous cell carcinoma | Autologous or allogeneic limbal epithelial stem cells expanded ex vivo on human amniotic membrane | Orphan Designation | - |
| neuroacanthocytosis | Bio-engineered oral mucosal tissue | Orphan Designation | - |
| ocular melanoma | Autologous or allogeneic limbal epithelial stem cells expanded ex vivo on human amniotic membrane | Orphan Designation | - |
| ocular surface squamous neoplasia | Autologous or allogeneic limbal epithelial stem cells expanded ex vivo on human amniotic membrane | Orphan Designation | - |
| spina bifida | cryopreserved human umbilical cord | Orphan Designation | - |