1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Kearns-Sayre syndrome | Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop-1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate | Orphan Designation | - |
| mitochondrial myopathy-cerebellar ataxia-pigmentary retinopathy syndrome | Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop-1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop-1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate | Orphan Designation | - |