2
Orphan Designations
1
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Azedra Ultratrace | Orphan Designation | - |
| Pelger-Huet anomaly | Methylnaltrexone | Orphan Designation | - |
| Schimke immuno-osseous dysplasia | Methylnaltrexone | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Azedra Ultratrace | Orphan Designation | - |
| cutaneous neuroendocrine carcinoma | Azedra Ultratrace | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Azedra Ultratrace | Orphan Designation | - |
| mucopolysaccharidosis type 6 | Methylnaltrexone | Orphan Designation | - |
| pancreatic neuroendocrine neoplasm | Azedra Ultratrace | Orphan Designation | - |
| pancreatic neuroendocrine tumor | Azedra Ultratrace | Orphan Designation | - |