2
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Aphexda | Orphan Designation | - |
| acute myeloid leukemia | motixafortide | Orphan Designation | - |
| anosmia for isobutyric acid | Aphexda | Orphan Designation | - |
| carcinoma in situ of urethra | Aphexda | Orphan Designation | - |
| double nail for fifth toe | Aphexda | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Aphexda | Orphan Designation | - |
| in situ carcinoma | Aphexda | Orphan Designation | - |
| neuroacanthocytosis | Aphexda | Orphan Designation | - |
| skin carcinoma in situ | Aphexda | Orphan Designation | - |
| stomach carcinoma in situ | Aphexda | Orphan Designation | - |
| urethral obstruction sequence | Aphexda | Orphan Designation | - |