1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Hurler syndrome | Sleeping Beauty Transposon-Engineered Autologous Plasmablasts for Expression and Delivery of Alpha-L-Iduronidase | Orphan Designation | - |
| Hurler-Scheie syndrome | Sleeping Beauty Transposon-Engineered Autologous Plasmablasts for Expression and Delivery of Alpha-L-Iduronidase | Orphan Designation | - |
| Scheie syndrome | Sleeping Beauty Transposon-Engineered Autologous Plasmablasts for Expression and Delivery of Alpha-L-Iduronidase | Orphan Designation | - |