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Company Daiichi Sankyo Co. Ltd.
DescriptionEthylene-bridged nucleic acid (ENA) oligonucleotide that induces exon 45 skipping of dystrophin mRNA
Molecular Target Dystrophin
Mechanism of Action 
Therapeutic ModalityNucleic acid
Latest Stage of DevelopmentPhase I/II
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation

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