1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Char syndrome | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |
| central nervous system cancer | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |
| mismatch repair cancer syndrome 1 | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Human alpha 2,6 sialyltransferase adenoviral gene therapy | Orphan Designation | - |