1
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| RNU5B-1 related neurodevelopmental disorder with seizures and joint laxity | Photofrin | Orphan Designation | - |
| developmental and epileptic encephalopathy 119 | Photofrin | Orphan Designation | - |
| leukodystrophy, hypomyelinating, 27 | Photofrin | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Photofrin | Orphan Designation | - |