3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| cerebellar ataxia | 2-amino-N-({methyl-[(6-trifluoromethoxy-benzothiazol-2-ylcarbamoyl)-methyl]-carbamoyl}-methyl)-acetamide monohydrochloride | Orphan Designation | - |
| multiple system atrophy | verdiperstat | Orphan Designation | - |
| multiple system atrophy, cerebellar type | verdiperstat | Orphan Designation | - |
| multiple system atrophy, parkinsonian type | verdiperstat | Orphan Designation | - |
| spinal muscular atrophy | Taldefgrobep Alfa | Orphan Designation | - |