2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Sandhoff disease | pyrimethamine | Orphan Designation | - |
| Tay-Sachs disease | pyrimethamineN-acetyl-glucosamine thiazoline | Orphan Designation | - |
| Tay-Sachs disease, B variant, adult form | pyrimethamineN-acetyl-glucosamine thiazoline | Orphan Designation | - |
| Tay-Sachs disease, B1 variant | pyrimethamine | Orphan Designation | - |
| Tay-Sachs disease, b variant, infantile form | pyrimethamine | Orphan Designation | - |
| Tay-Sachs disease, b variant, juvenile form | pyrimethamine | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | N-acetyl-glucosamine thiazoline | Orphan Designation | - |