2
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Diamond-Blackfan anemia | poloxamer 188 | Orphan Designation | - |
| Wilson disease | poloxamer 188 | Orphan Designation | - |
| acute chest syndrome | poloxamer 188 (purified) | Orphan Designation | - |
| hereditary persistence of fetal hemoglobin-sickle cell disease syndrome | poloxamer 188 (purified) | Orphan Designation | - |
| propionic acidemia | poloxamer 188 | Orphan Designation | - |
| sickle cell disease | poloxamer 188 (purified) | Orphan Designation | - |
| sickle cell-hemoglobin c disease syndrome | poloxamer 188 (purified) | Orphan Designation | - |
| sickle cell-hemoglobin d disease syndrome | poloxamer 188 (purified) | Orphan Designation | - |