1
Orphan Designations
1
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Mowat-Wilson syndrome | Cuvrior | Orphan Designation | - |
| Wilson disease | Cuvrior | Orphan Designation | - |
| Wilson-Turner syndrome | Cuvrior | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Cuvrior | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Cuvrior | Orphan Designation | - |
| primary adult heart tumor | Cuvrior | Orphan Designation | - |