7
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Combarros Calleja Leno syndrome | ganaxolone | Orphan Designation | - |
| Lennox-Gastaut syndrome | ganaxolone | Orphan Designation | - |
| annular epidermolytic ichthyosis | Ztalmy | Orphan Designation | - |
| cyclic hematopoiesis | Ztalmy | Orphan Designation | - |
| cyclic vomiting syndrome | Ztalmy | Orphan Designation | - |
| febrile seizures, familial | Ztalmy | Orphan Designation | - |
| fragile X syndrome | ganaxolone | Orphan Designation | - |
| infantile spasms | Ganaxolone | Orphan Designation | - |
| menstrual cycle-dependent periodic fever | Ztalmy | Orphan Designation | - |
| new-onset refractory status epilepticus | ganaxolone | Orphan Designation | - |
| protocadherin 3 | ganaxolone | Orphan Designation | - |
| thrombocytopenia, cyclic | Ztalmy | Orphan Designation | - |
| tuberous sclerosis | ganaxolone | Orphan Designation | - |