1
Orphan Designations
4
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| cystic kidney disease | Thiola | Orphan Designation | - |
| cystinosis | Thiola | Orphan Designation | - |
| hypotonia-cystinuria syndrome | Thiola | Orphan Designation | - |
| juvenile nephropathic cystinosis | Thiola | Orphan Designation | - |
| nephropathic cystinosis | Thiola | Orphan Designation | - |
| ventriculomegaly-cystic kidney disease | Thiola | Orphan Designation | - |