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eteplirsen (AVI-4658)

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Company Sarepta Therapeutics Inc.
DescriptionPhosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA
Molecular Target DNA
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentRegistration
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation U.S. - Fast Track (Treat Duchenne muscular dystrophy (DMD));
U.S. - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
U.S. - Priority Review (Treat Duchenne muscular dystrophy (DMD));
U.S. - Rare Pediatric Disease (Treat Duchenne muscular dystrophy (DMD));
EU - Orphan Drug (Treat Duchenne muscular dystrophy (DMD))
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