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SRP-4052

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Company Sarepta Therapeutics Inc.
DescriptionPhosphorodiamidate morpholino oligomer (PMO) targeting exon 52
Molecular Target DNA
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentPreclinical
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation
Partner

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