6
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Chondrocyte-alginate gel suspension | Orphan Designation | - |
| acute myeloid leukemia | (R)-N-(5-(3-hydroxypyrrolidin-1-yl)-2-morpholinooxazolo[4,5-b]pyridin-6-yl)-2-(2-methylpyridin-4-yl)oxazole-4-carboxamide | Orphan Designation | - |
| anosmia for isobutyric acid | Chondrocyte-alginate gel suspension | Orphan Designation | - |
| dermatomyositis | mesylate synthetic small molecule inhibitor of HDAC and PI3K | Orphan Designation | - |
| diffuse large B-cell lymphoma | C24H28N8O7S2 | Orphan Designation | - |
| liver diffuse large B-cell lymphoma | C24H28N8O7S2 | Orphan Designation | - |
| mevalonic aciduria | mesylate synthetic small molecule inhibitor of HDAC and PI3K | Orphan Designation | - |
| myelodysplastic syndrome | (R)-N-(5-(3-hydroxypyrrolidin-1-yl)-2-morpholinooxazolo[4,5-b]pyridin-6-yl)-2-(2-methylpyridin-4-yl)oxazole-4-carboxamide | Orphan Designation | - |
| neuroacanthocytosis | Chondrocyte-alginate gel suspension | Orphan Designation | - |
| nut midline carcinoma | mesylate synthetic small molecule inhibitor of HDAC and PI3K | Orphan Designation | - |
| primary central nervous system lymphoma | emavusertib | Orphan Designation | - |
| thyroid gland diffuse large B-cell lymphoma | C24H28N8O7S2 | Orphan Designation | - |