1
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | phoxillum | Orphan Designation | - |
| DYRK1A-related intellectual disability syndrome | phoxillum | Orphan Designation | - |
| anosmia for isobutyric acid | phoxillum | Orphan Designation | - |
| aortic valve stenosis | phoxillum | Orphan Designation | - |
| argininosuccinic aciduria | phoxillum | Orphan Designation | - |
| asthma, nasal polyps, and aspirin intolerance | phoxillum | Orphan Designation | - |
| double nail for fifth toe | phoxillum | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | phoxillum | Orphan Designation | - |
| neuroacanthocytosis | phoxillum | Orphan Designation | - |
| sudden unexpected infant death | phoxillum | Orphan Designation | - |