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Casimersen

Basics

Name

Casimersen

Pronunciation

(KAS i MER sen)

Brand Names: US

  • Amondys 45

Therapeutic Category

  • Antisense Oligonucleotide

Generic Availability (US)

Yes

Index Terms

  • Amondys 45

Clinical Pharmacology

Mechanism of Action

Binds to exon 45 of dystrophin premessenger RNA (mRNA), resulting in exclusion of this exon during mRNA processing. Exon 45 skipping allows for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping.

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