2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease | 2-(2-chlorobenzylidene)hydrazinecarboximidamide acetate | Orphan Designation | - |
| Charcot-Marie-Tooth disease-hearing loss-intellectual disability syndrome | 2-(2-chlorobenzylidene)hydrazinecarboximidamide acetate | Orphan Designation | - |
| amyotrophic lateral sclerosis | icerguastat | Orphan Designation | - |