1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| juvenile-onset Steinert myotonic dystrophy | a lyophilized modified oligonucleotide of 16 nucleotides conjugated to a molecule of oleic acid | Orphan Designation | - |
| late-onset Steinert myotonic dystrophy | a lyophilized modified oligonucleotide of 16 nucleotides conjugated to a molecule of oleic acid | Orphan Designation | - |
| myotonic dystrophy type 1 | a lyophilized modified oligonucleotide of 16 nucleotides conjugated to a molecule of oleic acid | Orphan Designation | - |