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Company Sarepta Therapeutics Inc.
DescriptionPhosphorodiamidate morpholino oligomer (PMO) targeting exon 53
Molecular Target
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentPhase III
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD); Treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping; Treat muscular dystrophy
Regulatory Designation

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