6
Orphan Designations
0
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CHIME syndrome | plasmin (human) | Orphan Designation | - |
| CINCA syndrome | Gamunex-C | Orphan Designation | - |
| chronic inflammatory demyelinating polyradiculoneuropathy | Gamunex-C | Orphan Designation | - |
| chronic relapsing inflammatory optic neuropathy | Gamunex-C | Orphan Designation | - |
| compensatory emphysema | alpha1 proteinase inhibitor (human) | Orphan Designation | - |
| cystic fibrosis | alpha1-proteinase inhibitor (human) | Orphan Designation | - |
| emphysema, hereditary pulmonary | alpha1 proteinase inhibitor (human) | Orphan Designation | - |
| familial partial lipodystrophy, Dunnigan type | plasmin (human) | Orphan Designation | - |
| familial thrombocytosis | plasmin (human) | Orphan Designation | - |
| myasthenia gravis | immune globulin (human) | Orphan Designation | - |
| neonatal inflammatory skin and bowel disease | Gamunex-C | Orphan Designation | - |
| polyneuropathy, inflammatory demyelinating, chronic | Gamunex-C | Orphan Designation | - |
| pulmonary emphysema | alpha1 proteinase inhibitor (human) | Orphan Designation | - |
| type 1 diabetes mellitus | alpha1-proteinase inhibitor (human) | Orphan Designation | - |
| type 1 diabetes mellitus 7 | alpha1-proteinase inhibitor (human) | Orphan Designation | - |
| type 1 diabetes mellitus 8 | alpha1-proteinase inhibitor (human) | Orphan Designation | - |