FDA Approves Second Exon-Skipping Drug for Duchenne | CheckRare.com

The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with certain dystrophin gene mutations have a dystrophin protein that is more functional than their …

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