3
Orphan Designations
1
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | Duvyzat | Orphan Designation | - |
| Duchenne muscular dystrophy | Duvyzat | Orphan Designation | - |
| acquired polycythemia vera | Givinostat | Orphan Designation | - |
| amyotrophic lateral sclerosis | Tiglutik | Orphan Designation | - |
| polycythemia | Givinostat | Orphan Designation | - |
| polycythemia neonatorum | Givinostat | Orphan Designation | - |
| symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers | Duvyzat | Orphan Designation | - |