4
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Aarskog-Scott syndrome, X-linked | N-acetylprocainamide | Orphan Designation | - |
| Emanuel syndrome | Bethanidine sulfate | Orphan Designation | - |
| Gitelman syndrome | Bethanidine sulfate | Orphan Designation | - |
| Skraban-Deardorff syndrome | Bethanidine sulfate | Orphan Designation | - |
| benign neoplasm of brain stem | Adenosine | Orphan Designation | - |
| congenital contractural arachnodactyly | N-acetylprocainamide | Orphan Designation | - |
| familial cutaneous collagenoma | Bethanidine sulfate | Orphan Designation | - |
| infratentorial cancer | Adenosine | Orphan Designation | - |
| infratentorial neoplasm | Adenosine | Orphan Designation | - |
| monosomy X | N-acetylprocainamide | Orphan Designation | - |
| myofibrillar myopathy 1 | Bethanidine sulfate | Orphan Designation | - |