5
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Huntington disease | small molecule inhibitor of phosphodiesterase 10 | Orphan Designation | - |
| MOGS-congenital disorder of glycosylation | human monoclonal antibody inhibitor of mannan binding lectin-associated serine protease-2 | Orphan Designation | - |
| central nervous system hematopoietic neoplasm | Yartemlea | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Yartemlea | Orphan Designation | - |
| disease related to hematopoietic stem cell transplant | Yartemlea | Orphan Designation | - |
| hematologic disorder | Yartemlea | Orphan Designation | - |
| hematopoietic and lymphoid cell neoplasm | Yartemlea | Orphan Designation | - |
| hematopoietic and lymphoid system neoplasm | Yartemlea | Orphan Designation | - |
| paroxysmal nocturnal hemoglobinuria | Humanized immunoglobulin G4 monoclonal antibody directed against mannan-binding lectin-associated serine protease-3 (MASP-3) | Orphan Designation | - |
| simple cryoglobulinemia | human monoclonal antibody inhibitor of mannan binding lectin-associated serine protease-2 | Orphan Designation | - |
| visceral neuropathy, familial, 1, autosomal recessive | human monoclonal antibody inhibitor of mannan binding lectin-associated serine protease-2 | Orphan Designation | - |