3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
8
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | recombinant AAV8 that contains a vector genome encoding a miniaturized dystrophin protein (microdystrophin) | Orphan Designation | - |
| Hurler syndrome | aden-associated virus vector serotype 9 expressing human a-L-iduronidase | Orphan Designation | - |
| Hurler-Scheie syndrome | aden-associated virus vector serotype 9 expressing human a-L-iduronidase | Orphan Designation | - |
| Scheie syndrome | aden-associated virus vector serotype 9 expressing human a-L-iduronidase | Orphan Designation | - |
| mucopolysaccharidosis type 2 | non-replicating recombinant adeno-associated virus (AAV) vector of serotype 9 expressing human iduronate-2-sulfatase | Orphan Designation | - |