2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| carbon monoxide-induced delayed encephalopathy | recombinant neuroglobin (rNgb) containing mutations H64Q/C46G/C55S/C120S | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | recombinant neuroglobin (rNgb) containing mutations H64Q/C46G/C55S/C120S | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | recombinant neuroglobin (rNgb) containing mutations H64Q/C46G/C55S/C120S | Orphan Designation | - |
| neuroblastoma | DTPA[Indium-111]-dinutuximab-IRDye800CW | Orphan Designation | - |