5
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | AlphaNine | Orphan Designation | - |
| Fanconi anemia complementation group D2 | Fibrinogen (human) | Orphan Designation | - |
| angioedema | C1 esterase inhibitor (human) | Orphan Designation | - |
| anosmia for isobutyric acid | AlphaNine | Orphan Designation | - |
| beta thalassemia | Isobutyramide | Orphan Designation | - |
| developmental and epileptic encephalopathy, 36 | AlphaNine | Orphan Designation | - |
| double nail for fifth toe | AlphaNine | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | AlphaNine | Orphan Designation | - |
| ischemic bowel disorder | Prostaglandin E1 in lipid emulsion | Orphan Designation | - |
| ischemic disease | Prostaglandin E1 in lipid emulsion | Orphan Designation | - |
| ischemic neuropathy | Prostaglandin E1 in lipid emulsion | Orphan Designation | - |
| neuroacanthocytosis | AlphaNine | Orphan Designation | - |
| sickle cell disease | Isobutyramide | Orphan Designation | - |