Eteplirsen, a novel drug for the treatment of DMD
YAO Jie
Abstract：Eteplirsen,a morpholino phosphorodiamidate oligonuclcotide,forces the exclusion of exon 51 from the mRNA of dystrophin protein,which induces dystrophin production.As a result,Duchenne muscular dystrophy (DMD),a fatal disease,will turn into Becker muscular dystrophy (BMD),a milder form of muscular dystrophy.In vitro assays and in vivo studies showed that eteplirsen is well tolerated and has mild side effects.Although under fire,eteplirsen has been approved by FDA in September 2016.We summarized and analyzed its pharmacological effect,pharmacokinetics,clinical use,and safety.
Keywords：Duchenne muscular dystrophyrare diseaseexon skipping therapymorpholino phosphorodiamidate oligonuclcotidesdystrophineteplirsen
Publication Date：2017-01-01
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：4( 2652-2655 )