1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Hurler syndrome | a single-stranded adeno-associated virus serotype 8 gene therapy containing codon optimized human alpha-1-iduronidase cDNA. | Orphan Designation | - |
| Hurler-Scheie syndrome | a single-stranded adeno-associated virus serotype 8 gene therapy containing codon optimized human alpha-1-iduronidase cDNA. | Orphan Designation | - |
| Scheie syndrome | a single-stranded adeno-associated virus serotype 8 gene therapy containing codon optimized human alpha-1-iduronidase cDNA. | Orphan Designation | - |