2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Dravet syndrome | 18-mer antisense oligonucleotide complementary to SCN1A mRNA | Orphan Designation | - |
| autosomal dominant optic atrophy | 18?mer antisense oligonucleotide complementary to OPA1 pre-mRNA | Orphan Designation | - |