3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acral dystrophic epidermolysis bullosa | autologous genetically modified human dermal fibroblasts | Orphan Designation | - |
| centripetalis recessive dystrophic epidermolysis bullosa | autologous genetically modified human dermal fibroblasts | Orphan Designation | - |
| hereditary hemochromatosis | Lentiviral vector expressing human fumarylacetoacetate hydrolase | Orphan Designation | - |
| hereditary hemorrhagic telangiectasia | Lentiviral vector expressing human fumarylacetoacetate hydrolase | Orphan Designation | - |
| hereditary spastic paraplegia | Lentiviral vector expressing human fumarylacetoacetate hydrolase | Orphan Designation | - |
| localized scleroderma | HDF-LV-RTS-MMP1 (human dermal fibroblasts genetically modified with LV-RTS-MMP1) and Veledimex | Orphan Designation | - |
| pretibial dystrophic epidermolysis bullosa | autologous genetically modified human dermal fibroblasts | Orphan Designation | - |