4
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Machado-Joseph disease | trehalose | Orphan Designation | - |
| adult-onset autosomal dominant demyelinating leukodystrophy | trehalose | Orphan Designation | - |
| amyotrophic lateral sclerosis | trehalose | Orphan Designation | - |
| distal 10q deletion syndrome | trehalose | Orphan Designation | - |
| oculopharyngeal muscular dystrophy | trehalose | Orphan Designation | - |
| oculopharyngeal muscular dystrophy 1 | trehalose | Orphan Designation | - |
| oculopharyngeal muscular dystrophy 2 | trehalose | Orphan Designation | - |