2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| glioma susceptibility 1 | [4-[(2R)-3-(3,4-dihydro-1H-isoquinolin-2-yl)-2-hydroxy-propyl]-8-[(1-methyl-4-piperidyl)oxy]-2,3-dihydro-1,4-benzoxazepin-5-one] | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | carisbamate | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | carisbamate | Orphan Designation | - |