3
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM1 gangliosidosis | biphenyl-substituted L-ido configured deoxynojirimycin derivative or Fluorobiphenyl-Piperidinetriol | Orphan Designation | - |
| Niemann-Pick disease type C | biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Sandhoff disease | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Tay-Sachs disease | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Tay-Sachs disease, B variant, adult form | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Tay-Sachs disease, B1 variant | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Tay-Sachs disease, b variant, infantile form | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |
| Tay-Sachs disease, b variant, juvenile form | Biphenyl-substituted L-ido configured deoxynojirimycin derivative | Orphan Designation | - |