1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| symptomatic form of Coffin-Lowry syndrome in female carriers | hydroxyurea | Orphan Designation | - |
| symptomatic form of fragile X syndrome in female carrier | hydroxyurea | Orphan Designation | - |
| symptomatic form of hemophilia A in female carriers | hydroxyurea | Orphan Designation | - |