4
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Olmsted syndrome | (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone(R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone | Orphan Designation | - |
| hereditary hemochromatosis | antagonist of the transient receptor potential cation channel, subfamily V, member 3 (TRPV3) | Orphan Designation | - |
| hereditary hemorrhagic telangiectasia | antagonist of the transient receptor potential cation channel, subfamily V, member 3 (TRPV3) | Orphan Designation | - |
| hereditary spastic paraplegia | antagonist of the transient receptor potential cation channel, subfamily V, member 3 (TRPV3) | Orphan Designation | - |
| pachyonychia congenita | (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone | Orphan Designation | - |