1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| juvenile-onset Steinert myotonic dystrophy | an antibody-siRNA conjugate, comprised of a humanized anti-TfR1 IgG1 monoclonal antibody covalently linked via a bis-maleimide non-cleavable linker to a double-stranded small interfering RNA directed to DMPK (myotonic dystrophy protein kinase) mRNA | Orphan Designation | - |
| late-onset Steinert myotonic dystrophy | an antibody-siRNA conjugate, comprised of a humanized anti-TfR1 IgG1 monoclonal antibody covalently linked via a bis-maleimide non-cleavable linker to a double-stranded small interfering RNA directed to DMPK (myotonic dystrophy protein kinase) mRNA | Orphan Designation | - |
| myotonic dystrophy type 1 | an antibody-siRNA conjugate, comprised of a humanized anti-TfR1 IgG1 monoclonal antibody covalently linked via a bis-maleimide non-cleavable linker to a double-stranded small interfering RNA directed to DMPK (myotonic dystrophy protein kinase) mRNA | Orphan Designation | - |