3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| carbon monoxide-induced delayed encephalopathy | Pozelimab/Cemdisiran Combination | Orphan Designation | - |
| juvenile myasthenia gravis | cemdisiran | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | Pozelimab/Cemdisiran Combination | Orphan Designation | - |
| myasthenia gravis | cemdisiran | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Pozelimab/Cemdisiran Combination | Orphan Designation | - |
| paroxysmal nocturnal hemoglobinuria | cemdisiran | Orphan Designation | - |
| transient neonatal myasthenia gravis | cemdisiran | Orphan Designation | - |