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Synonyms: Amondys 45® | SRP-4045 | SRP4045
casimersen is an approved drug (FDA (2021))
Compound class:
Synthetic organic
Comment: Casimersen is an antisense phosphorodiamidate morpholino oligomer (PMO) that is approved to treat certain patients with Duchenne muscular dystrophy (DMD). It promotes functional dystrophin synthesis in patients who have dystrophin gene variants that are amenable to exon 45 skipping, that is sufficient to restore some muscle function and slow disease progression.
The INN record for casimersen provides chemical identity as all-P-ambo-[2',3'-azanediyl-P-(dimethylamino)-P,2',3'- trideoxy-2',3'-seco](2'-N→5')(C-A-A-T-G-C-C-A-T-C-C-T-G-G-A-G-T-T-C-C-T-G) 5'-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,Ndimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or full IUPAC descriptor. |
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Classification  |
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| Compound class | Synthetic organic |
| Approved drug? | Yes (FDA (2021)) |
| International Nonproprietary Names |
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| INN number | INN |
| 10354 | casimersen |
| Synonyms |
| Amondys 45® | SRP-4045 | SRP4045 |
| Database Links |
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| CAS Registry No. | 1422958-19-7 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL4297566 |
| DrugBank Ligand | DB14984 |
| GtoPdb PubChem SID | 440816813 |
| Search PubMed clinical trials | casimersen |
| Search PubMed titles | casimersen |
| Search PubMed titles/abstracts | casimersen |
