Description: Vyondys 53 is an antisense oligonucleotide used for the treatment of patients with Duchenne muscular dystrophy who have a established mutation of the gene named DMD that is amenable to exon-53 skipping. Vyondys 53 (Golodirsen) treatment uses Sarepta’s proprietary PMO (Phosphorodiamidate Morpholino Oligomer) chemistry and an exon-skipping technology to bind to exon-53 of dystrophin pre-mRNA, resulting in exclusion, or skipping, of this exon during processing of mRNA in those with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for creation of an internally truncated dystrophin protein.
Category: Health & Fitness
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