1
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolf-Hirschhorn syndrome | Exisulind | Orphan Designation | - |
| congenital muscular dystrophy with intellectual disability and severe epilepsy | Exisulind | Orphan Designation | - |
| mismatch repair cancer syndrome 1 | Exisulind | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Exisulind | Orphan Designation | - |
| neuromuscular disease and ocular or auditory anomalies with or without seizures | Exisulind | Orphan Designation | - |