1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| central hypoventilation syndrome, congenital, 1, with or without Hirschsprung disease | a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous | Orphan Designation | - |
| oculodental syndrome, Rutherfurd type | a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous | Orphan Designation | - |
| primary failure of tooth eruption | a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous | Orphan Designation | - |