5
Orphan Designations
0
FDA Approvals
17
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GNE myopathy | aceneuramic acid | Orphan Designation | - |
| Tented eyebrows | sirolimus | Orphan Designation | - |
| facial dermoid cyst | Hyftor | Orphan Designation | - |
| facial nerve neoplasm | Hyftor | Orphan Designation | - |
| inherited Creutzfeldt-Jakob disease | (N,N¿-([cyclohexylmethylene]di-4,1-phenylene)bis(2-[1-pyrrolidinyl]acetamide) dimaleate | Orphan Designation | - |
| juvenile nasopharyngeal angiofibroma | Hyftor | Orphan Designation | - |
| kaposiform hemangioendothelioma | sirolimus | Orphan Designation | - |
| kaposiform lymphangiomatosis | sirolimus | Orphan Designation | - |
| lymphatic malformation | sirolimus | Orphan Designation | - |
| lymphatic malformation 5 | sirolimus | Orphan Designation | - |
| lymphatic malformation 6 | sirolimus | Orphan Designation | - |
| persistent idiopathic facial pain | Hyftor | Orphan Designation | - |
| spastic ataxia-corneal dystrophy syndrome | sirolimus | Orphan Designation | - |
| tuberous sclerosis 1 | Hyftor | Orphan Designation | - |
| tuberous sclerosis 2 | Hyftor | Orphan Designation | - |
| tufted angioma | sirolimus | Orphan Designation | - |
| woolly hair-skin fragility syndrome | sirolimus | Orphan Designation | - |