2
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Houge-Janssens syndrome 4 | Sabril | Orphan Designation | - |
| classic congenital adrenal hyperplasia | humanized anti-adrenocorticotropic hormone immunoglobulin gamma subclass 1 monoclonal antibody (anti-ACTH IgG1 mAb) | Orphan Designation | - |
| congenital adrenal hyperplasia | humanized anti-adrenocorticotropic hormone immunoglobulin gamma subclass 1 monoclonal antibody (anti-ACTH IgG1 mAb) | Orphan Designation | - |
| infantile spasms | Sabril | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | Sabril | Orphan Designation | - |
| mitochondrial DNA depletion syndrome, encephalomyopathic form with methylmalonic aciduria | Sabril | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Sabril | Orphan Designation | - |
| non-classic congenital adrenal hyperplasia | humanized anti-adrenocorticotropic hormone immunoglobulin gamma subclass 1 monoclonal antibody (anti-ACTH IgG1 mAb) | Orphan Designation | - |
| xeroderma pigmentosum group G | Sabril | Orphan Designation | - |