1
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Pelizaeus-Merzbacher disease in female carriers | Rivfloza | Orphan Designation | - |
| monosomy X | Rivfloza | Orphan Designation | - |
| mosaic monosomy X | Rivfloza | Orphan Designation | - |
| primary hyperoxaluria | Rivfloza | Orphan Designation | - |