3
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolf-Hirschhorn syndrome | Butyrylcholinesterase | Orphan Designation | - |
| autoimmune disorder of the nervous system | Butyrylcholinesterase | Orphan Designation | - |
| ciliary dyskinesia, primary, 41 | Butyrylcholinesterase | Orphan Designation | - |
| ciliary dyskinesia, primary, 47, and lissencephaly | Butyrylcholinesterase | Orphan Designation | - |
| congenital arteriovenous fistula | Allogeneic human aortic endothelial cells cultured in a porcine gelatin matrix | Orphan Designation | - |
| congenital systemic arteriovenous fistula | Allogeneic human aortic endothelial cells cultured in a porcine gelatin matrix | Orphan Designation | - |
| dural sinus malformation | Allogeneic human aortic endothelial cells cultured in a porcine gelatin matrix | Orphan Designation | - |
| post-surgical hypoinsulinemia | Butyrylcholinesterase | Orphan Designation | - |
| post-traumatic stress disorder | Butyrylcholinesterase | Orphan Designation | - |
| postsurgical hypothyroidism | Butyrylcholinesterase | Orphan Designation | - |
| primary ciliary dyskinesia 18 | Butyrylcholinesterase | Orphan Designation | - |
| primary ciliary dyskinesia 28 | Butyrylcholinesterase | Orphan Designation | - |