1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Tessadori-Van Haaften neurodevelopmental syndrome 3 | Genetically engineered (alpha-1,3-galactosyltransferase knock-out}, live cell porcine (Sus scrofa) xenotransplantation skin graft | Orphan Designation | - |
| intellectual developmental disorder, X-linked, syndromic, with pigmentary mosaicism and coarse facies | Genetically engineered (alpha-1,3-galactosyltransferase knock-out}, live cell porcine (Sus scrofa) xenotransplantation skin graft | Orphan Designation | - |
| muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1 | Genetically engineered (alpha-1,3-galactosyltransferase knock-out}, live cell porcine (Sus scrofa) xenotransplantation skin graft | Orphan Designation | - |