1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| intermediate maple syrup urine disease | recombinant AAV9 vector expressing functional, codon-optimized, human BCKDHA and BCKDHB genes which encode for catalytic E1alpha and E1beta subunits, respectively, of the BCKDH holoenzyme | Orphan Designation | - |
| intermittent maple syrup urine disease | recombinant AAV9 vector expressing functional, codon-optimized, human BCKDHA and BCKDHB genes which encode for catalytic E1alpha and E1beta subunits, respectively, of the BCKDH holoenzyme | Orphan Designation | - |
| maple syrup urine disease | recombinant AAV9 vector expressing functional, codon-optimized, human BCKDHA and BCKDHB genes which encode for catalytic E1alpha and E1beta subunits, respectively, of the BCKDH holoenzyme | Orphan Designation | - |