2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease type 4B3 | AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequence | Orphan Designation | - |
| Charcot-Marie-Tooth disease type 4F | AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequence | Orphan Designation | - |
| Charcot-Marie-Tooth disease type 4J | AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequence | Orphan Designation | - |
| aldosterone-producing adenoma with seizures and neurological abnormalities | Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 (melpida) | Orphan Designation | - |
| neurodevelopmental disorder with intracranial hemorrhage, seizures, and spasticity | Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 (melpida) | Orphan Designation | - |
| neurodevelopmental disorder with seizures, microcephaly, and brain abnormalities | Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 (melpida) | Orphan Designation | - |