4
Orphan Designations
4
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Fanconi anemia complementation group I | midazolam | Orphan Designation | - |
| Riley-Day syndrome | midazolam | Orphan Designation | - |
| ceroid lipofuscinosis, neuronal, 6B (Kufs type) | SEIZALAM | Orphan Designation | - |
| developmental and epileptic encephalopathy, 14 | SEIZALAM | Orphan Designation | - |
| ectodermal dysplasia syndrome | diazepam auto-injector | Orphan Designation | - |
| febrile infection-related epilepsy syndrome | SEIZALAM | Orphan Designation | - |
| febrile seizures, familial | midazolam | Orphan Designation | - |
| nerve agent poisoning | midazolam | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 10 | SEIZALAM | Orphan Designation | - |
| new-onset refractory status epilepticus | SEIZALAM | Orphan Designation | - |