2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Noonan syndrome 2 | varespladib sodium | Orphan Designation | - |
| Shwachman-Diamond syndrome 1 | varespladib sodium | Orphan Designation | - |
| acute chest syndrome | varespladib sodium | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | blisibimod | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | blisibimod | Orphan Designation | - |
| thrombocytopenia-absent radius syndrome | blisibimod | Orphan Designation | - |