2
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Cyklokapron | Orphan Designation | - |
| anosmia for isobutyric acid | Cyklokapron | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Cyklokapron | Orphan Designation | - |
| double nail for fifth toe | Cyklokapron | Orphan Designation | - |
| follicular lymphoma | Prednimustine | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Cyklokapron | Orphan Designation | - |
| large-cell immunoblastic lymphoma | Prednimustine | Orphan Designation | - |
| lymphoma | Prednimustine | Orphan Designation | - |
| neuroacanthocytosis | Cyklokapron | Orphan Designation | - |