4
Orphan Designations
0
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Blount disease, adolescent | RUCONEST | Orphan Designation | - |
| acute disseminated encephalomyelitis | RUCONEST | Orphan Designation | - |
| acute flaccid myelitis | RUCONEST | Orphan Designation | - |
| acute lymphoblastic leukemia | RUCONEST | Orphan Designation | - |
| adolescent idiopathic scoliosis | RUCONEST | Orphan Designation | - |
| angioedema | recombinant human C1-esterase inhibitor | Orphan Designation | - |
| benign focal seizures of adolescence | RUCONEST | Orphan Designation | - |
| capillary infantile hemangioma | recombinant human C1 inhibitor | Orphan Designation | - |
| capillary leak syndrome | recombinant human C1 inhibitor | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | recombinant human C1 inhibitor | Orphan Designation | - |
| cramps, familial adolescent | RUCONEST | Orphan Designation | - |
| delayed membranous cranial ossification | recombinant human C1 inhibitor | Orphan Designation | - |
| delayed puberty, self-limited | recombinant human C1 inhibitor | Orphan Designation | - |
| essential thrombocythemia | RUCONEST | Orphan Designation | - |
| eyelid capillary hemangioma | recombinant human C1 inhibitor | Orphan Designation | - |
| familial thrombocytosis | RUCONEST | Orphan Designation | - |