2
Orphan Designations
1
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| autosomal recessive multiple pterygium syndrome | NexoBrid | Orphan Designation | - |
| dilated cardiomyopathy | ixmyelocel-T | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | NexoBrid | Orphan Designation | - |
| pediatric hepatocellular carcinoma | NexoBrid | Orphan Designation | - |
| pediatric ovarian dysgerminoma | NexoBrid | Orphan Designation | - |
| pediatric ovarian germ cell tumor | NexoBrid | Orphan Designation | - |
| pediatric-onset Graves disease | NexoBrid | Orphan Designation | - |
| thickened earlobes-conductive deafness syndrome | NexoBrid | Orphan Designation | - |