2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Down syndrome | A recombinant adeno-associated virus (rAAV) vector-based gene therapy expressing the methylmalonyl-CoA mutase (MMUT) transgene | Orphan Designation | - |
| Matthew-Wood syndrome | A recombinant adeno-associated virus (rAAV) vector-based gene therapy expressing the methylmalonyl-CoA mutase (MMUT) transgene | Orphan Designation | - |
| chromosome 15q26-qter deletion syndrome | A recombinant adeno-associated virus (rAAV) vector-based gene therapy expressing the methylmalonyl-CoA mutase (MMUT) transgene | Orphan Designation | - |
| methylmalonic acidemia | hydroxocobalamin and methylcobalamin | Orphan Designation | - |