4
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Nager acrofacial dysostosis | Recombinant human histidyl-tRNA synthetase | Orphan Designation | - |
| Peters plus syndrome | Recombinant human histidyl-tRNA synthetase | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy | recombinant human histidyl tRNA synthetase | Orphan Designation | - |
| mitochondrial DNA deletion syndrome with progressive myopathy | Recombinant human histidyl-tRNA synthetase | Orphan Designation | - |
| sarcoidosis | Human IgG1Fc- iMod domain (amino acids 2-60) of human HARS fusion protein | Orphan Designation | - |
| systemic sclerosis | efzofitimod | Orphan Designation | - |