7
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Stargardt disease | lentiviral vector containing the human ABCA4 gene | Orphan Designation | - |
| autoimmune hemolytic anemia | rilzabrutinib | Orphan Designation | - |
| classic phenylketonuria | Adeno-associated virus (AAV)-based vector with an engineered capsid serotype SNY001 harboring human phenylalanine hydroxylase (hPAH) cDNA | Orphan Designation | - |
| immunoglobulin G4-related sclerosing disease | rilzabrutinib | Orphan Designation | - |
| immunoglobulin M, level of | rilzabrutinib | Orphan Designation | - |
| immunoglobulin-mediated membranoproliferative glomerulonephritis | rilzabrutinib | Orphan Designation | - |
| mixed-type autoimmune hemolytic anemia | rilzabrutinib | Orphan Designation | - |
| myotonic dystrophy type 1 | a nonreplicating adeno-associated virus, serotype SAN011 that expresses an artificial microRNA | Orphan Designation | - |
| non-autoimmune hemolytic anemia | rilzabrutinib | Orphan Designation | - |
| phenylketonuria | Adeno-associated virus (AAV)-based vector with an engineered capsid serotype SNY001 harboring human phenylalanine hydroxylase (hPAH) cDNA | Orphan Designation | - |
| phenylketonuria | Adeno-associated virus (AAV)-based vector with an engineered capsid serotype SNY001 harboring human phenylalanine hydroxylase (hPAH) cDNA | Orphan Designation | - |
| retinitis pigmentosa | lentiviral vector containing the human MY07A gene | Orphan Designation | - |
| sickle cell disease | rilzabrutinib | Orphan Designation | - |
| sickle cell-hemoglobin c disease syndrome | rilzabrutinib | Orphan Designation | - |
| sickle cell-hemoglobin d disease syndrome | rilzabrutinib | Orphan Designation | - |