3
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DPM3-congenital disorder of glycosylation | adeno-associated virus serotype 8 containing the human SGCG geneadeno-associated virus serotype 9/rh74 containing the human Capn3 gene and a target sequence of cardiac-specific microRNAAdeno-associated virus serotype 9 carrying the human Fukutin-Related protein gene and target sequence of the miR-208a | Orphan Designation | - |
| autosomal recessive spinocerebellar ataxia 2 | adeno-associated virus serotype 8 containing the human SGCG geneadeno-associated virus serotype 9/rh74 containing the human Capn3 gene and a target sequence of cardiac-specific microRNAAdeno-associated virus serotype 9 carrying the human Fukutin-Related protein gene and target sequence of the miR-208a | Orphan Designation | - |
| spinocerebellar ataxia, autosomal recessive 29 | adeno-associated virus serotype 8 containing the human SGCG geneadeno-associated virus serotype 9/rh74 containing the human Capn3 gene and a target sequence of cardiac-specific microRNAAdeno-associated virus serotype 9 carrying the human Fukutin-Related protein gene and target sequence of the miR-208a | Orphan Designation | - |