4
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Mowat-Wilson syndrome | modified human P-type copper transporting ATPase (hATP7B) messenger ribonucleic acid encapsulated in a lipid nanoparticle (hATP7B mRNA-LNP) | Orphan Designation | - |
| Wilson disease | modified human P-type copper transporting ATPase (hATP7B) messenger ribonucleic acid encapsulated in a lipid nanoparticle (hATP7B mRNA-LNP) | Orphan Designation | - |
| Wilson-Turner syndrome | modified human P-type copper transporting ATPase (hATP7B) messenger ribonucleic acid encapsulated in a lipid nanoparticle (hATP7B mRNA-LNP) | Orphan Designation | - |
| homocystinuria | Modified human cystathionine beta synthase messenger ribonucleic acid encapsulated in a lipid nanoparticle | Orphan Designation | - |
| progressive familial intrahepatic cholestasis | modified human ATP binding cassette subfamily B member 4 (ABCB4) mRNA encoding multidrug resistance protein 3 (MDR3)modified human ATP binding cassette subfamily B member 11 (ABCB11) messenger RNA encoding bile salts export pump | Orphan Designation | - |