7
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Ehlers-Danlos syndrome, vascular type | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| Gaucher disease | Zavesca | Orphan Designation | - |
| Koolen-de Vries syndrome due to 17q21.31 microdeletion syndrome | Zavesca | Orphan Designation | - |
| Koolen-de Vries syndrome due to a point mutation | Zavesca | Orphan Designation | - |
| autosomal dominant polycystic kidney disease | Menadione Sodium Bisulfitesodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| autosomal dominant polycystic liver disease | menadione sodium bisulfitesodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| metastatic malignant neoplasm in the lymph nodes | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| metastatic melanoma | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| metastatic neoplasm | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| metastatic squamous cell carcinoma | Sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Zavesca | Orphan Designation | - |
| noninfectious dermatoses of eyelid | sodium ascorbate and menadione sodium bisulfite | Orphan Designation | - |