1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute chest syndrome | Siklos | Orphan Designation | - |
| hereditary persistence of fetal hemoglobin-sickle cell disease syndrome | Siklos | Orphan Designation | - |
| sickle cell disease | Siklos | Orphan Designation | - |
| sickle cell-hemoglobin c disease syndrome | Siklos | Orphan Designation | - |
| sickle cell-hemoglobin d disease syndrome | Siklos | Orphan Designation | - |
| syndromic X-linked intellectual disability 5 | Siklos | Orphan Designation | - |