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The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 2020;30:67-70   Introduction On December 12, 2019, the Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics for golodirsen to treat Duchenne muscular dystrophy (DMD) patients with eligible mutations (www.drugs.com/newdrugs/fda-approves-vyondys-53-golodirsen-duchenne-muscular-dystrophy-dmd-patients-amenable-skipping-exon-5119.html). This brings the number of FDA […]