For research use only. Not for therapeutic Use.
Casimersen sodium (Cat No.: I040119) is an antisense oligonucleotide therapy developed to treat Duchenne muscular dystrophy (DMD), specifically targeting exon 45 of the dystrophin gene. By promoting exon skipping during mRNA splicing, casimersen allows the production of a shortened but functional form of dystrophin, which is critical for muscle cell stability. This approach helps improve muscle function and slows disease progression in patients with DMD who have specific mutations amenable to exon 45 skipping. Casimersen sodium is a targeted, gene-modulating treatment for DMD.
Molecular Formula | C268H402N124Na22O95P22 |
Purity | ≥95% |
Reference | [1]. Shirley M. Casimersen: First Approval. Drugs. 2021;81(7):875-879. [2]. Casimersen. Am J Health Syst Pharm. 2021;78(13):1149-1150. |