3
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Waardenburg syndrome | Recombinant retroviral vector - glucocerebrosidase | Orphan Designation | - |
| alpha-mannosidosis | Recombinant retroviral vector - glucocerebrosidase | Orphan Designation | - |
| congenital disorder of glycosylation, type IIw | Recombinant retroviral vector - glucocerebrosidase | Orphan Designation | - |
| cystic fibrosis | Cystic fibrosis transmembrane conductance regulator gene | Orphan Designation | - |
| metastatic malignant neoplasm in the brain | Herpes simplex virus gene | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Herpes simplex virus gene | Orphan Designation | - |
| metastatic melanoma | Herpes simplex virus gene | Orphan Designation | - |
| primary biliary cholangitis | Herpes simplex virus gene | Orphan Designation | - |
| primary cutaneous lymphoma | Herpes simplex virus gene | Orphan Designation | - |
| primary hyperoxaluria | Herpes simplex virus gene | Orphan Designation | - |