6
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| bronchiectasis | Alpha1-antitrypsin(human) | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | alpha-1 proteinase inhibitor (human) | Orphan Designation | - |
| congenital bile acid synthesis defect 1 | Alpha1-Proteinase Inhibitor (Human) | Orphan Designation | - |
| congenital disorder of deglycosylation 1 | Alpha1-Proteinase Inhibitor (Human) | Orphan Designation | - |
| cystic fibrosis | Alpha1-Proteinase Inhibitor (Human) | Orphan Designation | - |
| graft versus host disease | alpha-1 proteinase inhibitor (human) | Orphan Designation | - |
| isolated thyroid-stimulating hormone deficiency | Alpha1-Proteinase Inhibitor (Human) | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | alpha-1 proteinase inhibitor (human) | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | alpha-1 proteinase inhibitor (human) | Orphan Designation | - |