Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells

Abstract excerpt

BACKGROUND: Antisense oligonucleotides (AON) represent a promising treatment for Duchenne muscular dystrophy (DMD) carrying out-of-frame deletions, but also show limitations. In a completed clinical trial golodirsen, approved by FDA to induce skipping of DMD gene exon 53 in eligible patients, we demonstrated increase in DMD expression and protein production, albeit with inter-patient variability. METHODS: Here,...

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