1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM1 gangliosidosis | trisodium methane trisdiazeniumdiolate (MD3) | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 9 | trisodium methane trisdiazeniumdiolate (MD3) | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | trisodium methane trisdiazeniumdiolate (MD3) | Orphan Designation | - |