7
Orphan Designations
0
FDA Approvals
13
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM2 gangliosidosis | GemfibrozilTrans-Cinnamic Acid | Orphan Designation | - |
| Krabbe disease | GemfibrozilTrans-Cinnamic Acid | Orphan Designation | - |
| Sandhoff disease | Gemfibrozil | Orphan Designation | - |
| Tay-Sachs disease | Gemfibrozil | Orphan Designation | - |
| brachydactyly type B | Trans-Cinnamic Acid | Orphan Designation | - |
| congenital lethal erythroderma | Trans-Cinnamic Acid | Orphan Designation | - |
| malignant carotid body paraganglioma | Trans-Cinnamic Acid | Orphan Designation | - |
| neuronal ceroid lipofuscinosis | gemfibrozil and vitamin Agemfibrozil | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 1 | gemfibrozil and vitamin Agemfibrozil | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 3 | gemfibrozil and vitamin Agemfibrozil | Orphan Designation | - |
| post 5-alpha-reductase inhibitors treatment syndrome | Trans-Cinnamic Acid | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Trans-Cinnamic Acid | Orphan Designation | - |
| treatment-refractory schizophrenia | Trans-Cinnamic Acid | Orphan Designation | - |