2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| classic phenylketonuria | recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH) | Orphan Designation | - |
| homozygous familial hypercholesterolemia | recombinant adeno-associated virus serotype 8 vector encoding human low-density lipoprotein receptor | Orphan Designation | - |
| phenylketonuria | recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH) | Orphan Designation | - |
| phenylketonuria | recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH) | Orphan Designation | - |