3
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Ketoconazole | Orphan Designation | - |
| anosmia for isobutyric acid | Ketoconazole | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 1 | Papaverine topical gel | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 2 | Papaverine topical gel | Orphan Designation | - |
| autosomal dominant hypocalcemia 1 | Calcium acetate | Orphan Designation | - |
| hyperphosphatasia with intellectual disability syndrome 3 | Calcium acetate | Orphan Designation | - |
| neuroacanthocytosis | Ketoconazole | Orphan Designation | - |
| renal hypomagnesemia 3 | Calcium acetate | Orphan Designation | - |
| surfactant metabolism dysfunction, pulmonary, 5 | Papaverine topical gel | Orphan Designation | - |