Eteplirsen

• CAT Number: I045651
• CAS Number: 1173755-55-9
• Molecular Weight: 10306.00
• Purity: 90%
• Target: DMD mRNA
• Target Protein: P11532
• Appearance: Solid
• Storage: Dry, dark and at 2 - 8 °C for six months or -20°C for two years.

Eteplirsen（Cat No.:I045651）is an antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. It works by binding to exon 51 of the dystrophin pre-mRNA, enabling the production of a truncated but functional dystrophin protein. This helps slow the progression of muscle degeneration in DMD patients. Administered via intravenous infusion, Eteplirsen provides a targeted therapeutic approach that addresses the underlying genetic cause of DMD, improving mobility and quality of life in affected individuals.

Reference

[1]. Kenji Rowel Q Lim, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545.
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