8
Orphan Designations
2
FDA Approvals
24
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Photrexa Viscous | Orphan Designation | - |
| Bruck syndrome | mitomycin | Orphan Designation | - |
| Ramos-Arroyo syndrome | Antisense oligonucleotide directed against connexin43 | Orphan Designation | - |
| abetalipoproteinemia | Photrexa Viscous | Orphan Designation | - |
| acrofacial dysostosis Cincinnati type | mitomycin | Orphan Designation | - |
| anterior segment dysgenesis 8 | Antisense oligonucleotide directed against connexin43 | Orphan Designation | - |
| central corneal ulcer | riboflavin 5'-phosphate sodium | Orphan Designation | - |
| corneal ectasia | Photrexa Viscous | Orphan Designation | - |
| corneal ulcer | riboflavin 5'-phosphate sodium | Orphan Designation | - |
| dermochondrocorneal dystrophy | mitomycin | Orphan Designation | - |
| epithelial and subepithelial corneal dystrophy | mitomycin | Orphan Designation | - |
| has an isolated presentation | Mitosol | Orphan Designation | - |
| keratoconus | copper sulfatePhotrexa Viscous | Orphan Designation | - |
| laryngo-onycho-cutaneous syndrome | mitomycin | Orphan Designation | - |
| mycotic corneal ulcer | riboflavin 5'-phosphate sodium | Orphan Designation | - |
| persistent hyperplastic primary vitreous, autosomal recessive | Antisense oligonucleotide directed against connexin43 | Orphan Designation | - |
| progressive familial intrahepatic cholestasis | Photrexa Viscous | Orphan Designation | - |
| progressive supranuclear palsy | Photrexa Viscous | Orphan Designation | - |
| refractory celiac disease | Mitosol | Orphan Designation | - |
| refractory cytopenia of childhood | Mitosol | Orphan Designation | - |
| refractory hairy cell leukemia | Mitosol | Orphan Designation | - |
| refractory plasma cell neoplasm | Mitosol | Orphan Designation | - |
| subepithelial mucinous corneal dystrophy | mitomycin | Orphan Designation | - |
| testis refractory cancer | Mitosol | Orphan Designation | - |