golodirsen (Pending FDA Approval)

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Dosing & Uses

Duchenne Muscular Dystrophy

Pending FDA approval for Duchenne muscular dystrophy (DMD) with genetic mutations amenable to exon 53 skipping

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Pharmacology

Mechanism of Action

Phosphorodiamidate morpholino oligomer (PMO) that binds to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or skipping, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping

Exon skipping is intended to allow for production of an internally truncated, but functional dystrophin protein

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.