3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | Enhanced delivery peptide-oligonucleotide conjugate for Duchenne Muscular Dystrophy Exon 51 | Orphan Designation | - |
| juvenile-onset Steinert myotonic dystrophy | a synthetic peptide-oligonucleotide conjugate in which a phosphorodiamidate morpholino oligonucleotide is conjugated to a cell penetrating peptide | Orphan Designation | - |
| late-onset Steinert myotonic dystrophy | a synthetic peptide-oligonucleotide conjugate in which a phosphorodiamidate morpholino oligonucleotide is conjugated to a cell penetrating peptide | Orphan Designation | - |
| myotonic dystrophy type 1 | a synthetic peptide-oligonucleotide conjugate in which a phosphorodiamidate morpholino oligonucleotide is conjugated to a cell penetrating peptide | Orphan Designation | - |
| pediatric multiple sclerosis | recombinant ovine interferon tau | Orphan Designation | - |