2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease axonal type 2C | 1-({(5S,7S,8R)-8-fluoro-3-[5-(2-hydroxypropan-2-yl)pyrazin-2-yl]-7-methyl-2-oxo-1-oxa-3-azaspiro[4.5]decan-7-yl}methyl)-1H-1,3-benzimidazole-6-carbonitrile | Orphan Designation | - |
| developmental and epileptic encephalopathy, 14 | Anti-epileptic small molecule inhibitor of KCNT1 | Orphan Designation | - |