4
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| congenital lactic acidosis, Saguenay-Lac-Saint-Jean type | Sodium dichloroacetate | Orphan Designation | - |
| developmental and epileptic encephalopathy 119 | Sodium dichloroacetate | Orphan Designation | - |
| hereditary painful callosities | Fructose-1,6-diphosphate | Orphan Designation | - |
| hypotonia-failure to thrive-microcephaly syndrome | H.P. Acthar Gel | Orphan Designation | - |
| infantile spasms | H.P. Acthar Gel | Orphan Designation | - |
| insensitivity to pain with hyperplastic Myelinopathy | H.P. Acthar Gel | Orphan Designation | - |
| neurodevelopmental disorder with involuntary movements | Sodium dichloroacetate | Orphan Designation | - |
| neuropathy, painful | Fructose-1,6-diphosphate | Orphan Designation | - |
| twin to twin transfusion syndrome | H.P. Acthar Gel | Orphan Designation | - |