1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | rSP-C surfactant | Orphan Designation | - |
| anosmia for isobutyric acid | rSP-C surfactant | Orphan Designation | - |
| double nail for fifth toe | rSP-C surfactant | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | rSP-C surfactant | Orphan Designation | - |
| neuroacanthocytosis | rSP-C surfactant | Orphan Designation | - |
| severe Canavan disease | rSP-C surfactant | Orphan Designation | - |