4
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine | Orphan Designation | - |
| Ewing sarcoma | bi-shRNA furin and GMCSF Autologous Tumor Cell Vaccine | Orphan Designation | - |
| Finnish type amyloidosis | bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine | Orphan Designation | - |
| GNE myopathy | GNE Lipoplex | Orphan Designation | - |
| hereditary inclusion body myopathy-joint contractures-ophthalmoplegia syndrome | GNE Lipoplex | Orphan Designation | - |
| hereditary inclusion-body myopathy | GNE Lipoplex | Orphan Designation | - |
| lysinuric protein intolerance | bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine | Orphan Designation | - |
| ovarian cancer | bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine | Orphan Designation | - |