Golodirsen Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression in Duchenne Muscular Dystrophy Patients With Mutations Amenable to Exon 53 Skipping (S22.001) - Concepedia

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Golodirsen Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression in Duchenne Muscular Dystrophy Patients With Mutations Amenable to Exon 53 Skipping (S22.001)

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Citations

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References

2018

Year

Francesco Muntoni, Diane E. Frank, Valentina Sardone, J.E. Morgan, Fred Schnell, Jay S. Charleston, Cody A. Desjardins, Rahul Phadke, Caroline A. Sewry, Linda Popplewell,

Abstract

To conduct an analysis of exon 53 skipping and dystrophin production and its cellular localization in muscle biopsies from patients in an ongoing trial of golodirsen (formerly SRP-4053).