1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| African iron overload | (S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiaxolecarboxylic acid | Orphan Designation | - |
| GRACILE syndrome | (S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiaxolecarboxylic acid | Orphan Designation | - |
| neurodegeneration with brain iron accumulation 2A | (S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiaxolecarboxylic acid | Orphan Designation | - |