7
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease | Adeno-associated virus 9 vector expressing a functional human codon optimized cDNA encoding glucosylceramidase beta 1 | Orphan Designation | - |
| Leigh syndrome | adeno-associated virus 9 human NADH:ubiquinone oxidoreductase subunit S4 | Orphan Designation | - |
| Niemann-Pick disease type C | AAV9-hNPC1 is an Adeno-Associated Virus 9 (AAV9) vector expressing a human cDNA encoding Niemann-Pick C1 (NPC1), under control of the Elongation Factor 1 alpha Short (EF1S) promoter | Orphan Designation | - |
| methylmalonic acidemia | adeno-associated virus 9 methylmalonyl Co-A mutase (AAV9-MMUT)Adeno-Associated Virus 9 vector expressing human Metabolism of Cobalamin Associated C genehydroxocobalamin | Orphan Designation | - |
| methylmalonic acidemia with homocystinuria, type cblJ | methylcobalamin | Orphan Designation | - |
| methylmalonic aciduria and homocystinuria type cblF | methylcobalamin | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 9 | methylcobalamin | Orphan Designation | - |