4
Orphan Designations
3
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ABetaA21G amyloidosis | dichlorphenamide | Orphan Designation | - |
| Andersen-Tawil syndrome | dichlorphenamide | Orphan Designation | - |
| Kabuki syndrome 1 | glucagon infusion | Orphan Designation | - |
| MEGF10-related myopathy | dichlorphenamide | Orphan Designation | - |
| acute adrenal insufficiency | dichlorphenamide | Orphan Designation | - |
| acute flaccid myelitis | diazepam subcutaneous injection | Orphan Designation | - |
| acute lymphoblastic leukemia | diazepam subcutaneous injection | Orphan Designation | - |
| autosomal recessive osteopetrosis 3 | dichlorphenamide | Orphan Designation | - |
| cerebral amyloid angiopathy, APP-related | dichlorphenamide | Orphan Designation | - |
| febrile infection-related epilepsy syndrome | diazepam subcutaneous injection | Orphan Designation | - |
| hyperinsulinemic hypoglycemia | glucagon (ready-to-use) | Orphan Designation | - |
| hyperkalemic periodic paralysis | dichlorphenamide | Orphan Designation | - |
| liver disease, severe congenital | glucagon infusion | Orphan Designation | - |
| megalencephaly-severe kyphoscoliosis-overgrowth syndrome | glucagon infusion | Orphan Designation | - |
| primary biliary cholangitis | dichlorphenamide | Orphan Designation | - |
| pulmonary immaturity | dichlorphenamide | Orphan Designation | - |