1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Niemann-Pick disease type C | 2-hydroxypropyl-B-cyclodextrin | Orphan Designation | - |
| Niemann-Pick disease, type C1 | 2-hydroxypropyl-B-cyclodextrin | Orphan Designation | - |
| Niemann-Pick disease, type C2 | 2-hydroxypropyl-B-cyclodextrin | Orphan Designation | - |