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DYNE-251 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1). It is being investigated for the treatment of Duchenne muscular dystrophy (DMD) in patients who are amenable to exon 51 skipping.
DYNE-251 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1). It is being investigated for the treatment of Duchenne muscular dystrophy (DMD) in patients who are amenable to exon 51 skipping.
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