3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | [(4-benzylpiperazin-1-yl)(2-(isopentylamino)pyridin-3-yl)methanone]-phosphate | Orphan Designation | - |
| amyotrophic lateral sclerosis | human spinal cord derived neural stem cells | Orphan Designation | - |
| cutaneous neuroendocrine carcinoma | Seneca Valley virus | Orphan Designation | - |
| pancreatic neuroendocrine neoplasm | Seneca Valley virus | Orphan Designation | - |
| pancreatic neuroendocrine tumor | Seneca Valley virus | Orphan Designation | - |