1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Finnish type amyloidosis | mannopentaose phosphate sulfate | Orphan Designation | - |
| X-linked Alport syndrome | mannopentaose phosphate sulfate | Orphan Designation | - |
| high anorectal malformation | mannopentaose phosphate sulfate | Orphan Designation | - |
| high-grade B-cell lymphoma double-hit/triple-hit | mannopentaose phosphate sulfate | Orphan Designation | - |
| hyperopia, high | mannopentaose phosphate sulfate | Orphan Designation | - |
| severe nonproliferative diabetic retinopathy | mannopentaose phosphate sulfate | Orphan Designation | - |