1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult-onset progressive leukoencephalopathy-early-onset deafness | iluzanebart | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | iluzanebart | Orphan Designation | - |
| large congenital melanocytic nevus | iluzanebart | Orphan Designation | - |
| leukodystrophy, demyelinating, adult-onset, autosomal dominant, typical | iluzanebart | Orphan Designation | - |
| leukoencephalopathy, diffuse hereditary, with spheroids 1 | iluzanebart | Orphan Designation | - |
| muscular dystrophy, adult-onset, with leukoencephalopathy | iluzanebart | Orphan Designation | - |