1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Hurler syndrome | alpha-L-iduronidase (IDUA) - ricin transport subunit B (RTB) lectin protein fusion | Orphan Designation | - |
| Hurler-Scheie syndrome | alpha-L-iduronidase (IDUA) - ricin transport subunit B (RTB) lectin protein fusion | Orphan Designation | - |
| Scheie syndrome | alpha-L-iduronidase (IDUA) - ricin transport subunit B (RTB) lectin protein fusion | Orphan Designation | - |