4
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM2 gangliosidosis | venglustat malate | Orphan Designation | - |
| Kleefstra syndrome 1 | Nexviazyme | Orphan Designation | - |
| PDA1 | Nexviazyme | Orphan Designation | - |
| achondroplasia | Anti-fibroblast growth factor receptor 3 antigen-binding fragment (anti-FGFR3 Fab) | Orphan Designation | - |
| autosomal dominant polycystic kidney disease | venglustat | Orphan Designation | - |
| chromosome 17q23.1-q23.2 deletion syndrome | Nexviazyme | Orphan Designation | - |
| chromosome 1q21.1 deletion syndrome | Nexviazyme | Orphan Designation | - |
| glycogen storage disease II | Nexviazyme | Orphan Designation | - |
| restrictive dermopathy 1 | Nexviazyme | Orphan Designation | - |