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Golodirsen: First Approval

Heo YA. Golodirsen: First Approval. Drugs 2020;80:329-333   Abstract Golodirsen (Vyondys 53), an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO) subclass designed to induce exon 53 skipping, has been developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). In December 2019, intravenous golodirsen received its first global approval in the USA […]