2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wiskott-Aldrich syndrome | Waskyra | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Waskyra | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Waskyra | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Waskyra | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Waskyra | Orphan Designation | - |
| pediatric-onset Graves disease | Waskyra | Orphan Designation | - |
| severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency | autologus CD34+ cells transfected with retroviral vector containing adenosine deaminase gene | Orphan Designation | - |