1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Lafora disease | a therapeutic transgene cassette with an adeno-associated viral (AAV)-9 vector expressing a functional human codon optimized complimentary deoxyribonucleic acid (cDNA) encoding hEPM2A, under control of a CAG promoter | Orphan Designation | - |
| early-onset Lafora body disease | a therapeutic transgene cassette with an adeno-associated viral (AAV)-9 vector expressing a functional human codon optimized complimentary deoxyribonucleic acid (cDNA) encoding hEPM2A, under control of a CAG promoter | Orphan Designation | - |
| myoclonic epilepsy of Lafora 2 | a therapeutic transgene cassette with an adeno-associated viral (AAV)-9 vector expressing a functional human codon optimized complimentary deoxyribonucleic acid (cDNA) encoding hEPM2A, under control of a CAG promoter | Orphan Designation | - |