5
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| BH4-deficient hyperphenylalaninemia A | L-5-hydroxytryptophan | Orphan Designation | - |
| DYRK1A-related intellectual disability syndrome | Testosterone | Orphan Designation | - |
| anosmia for isobutyric acid | Testosterone | Orphan Designation | - |
| dihydropteridine reductase deficiency | L-5-hydroxytryptophan | Orphan Designation | - |
| fragile X syndrome | guanfacine | Orphan Designation | - |
| leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome | progesterone | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 11 | progesterone | Orphan Designation | - |
| mitochondrial DNA maintenance syndrome | progesterone | Orphan Designation | - |
| neuroacanthocytosis | Testosterone | Orphan Designation | - |
| pterin-4 alpha-carbinolamine dehydratase 1 deficiency | L-5-hydroxytryptophan | Orphan Designation | - |