4
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Modeyso | Orphan Designation | - |
| Sandhoff disease, adult form | Modeyso | Orphan Designation | - |
| acute myeloid leukemia | 2-O, 3-O, desulfated heparin | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Modeyso | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Modeyso | Orphan Designation | - |
| gastric mantle cell lymphoma | 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one-2HCL | Orphan Designation | - |
| glioblastoma | 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one*2HCl | Orphan Designation | - |
| malignant glioma | Modeyso | Orphan Designation | - |
| mantle cell lymphoma | 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one-2HCL | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Modeyso | Orphan Designation | - |
| splenic mantle cell lymphoma | 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one-2HCL | Orphan Designation | - |