2
Orphan Designations
1
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | DORC ILM-BlueMembraneBlue | Orphan Designation | - |
| anosmia for isobutyric acid | DORC ILM-BlueMembraneBlue | Orphan Designation | - |
| double nail for fifth toe | DORC ILM-BlueMembraneBlue | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | DORC ILM-BlueMembraneBlue | Orphan Designation | - |
| neuroacanthocytosis | DORC ILM-BlueMembraneBlue | Orphan Designation | - |
| selective intrauterine growth restriction | MembraneBlue | Orphan Designation | - |