descriptionPublicationkeyboard_double_arrow_right Article 01 Nov 2016 English Publisher:Elsevier BVJournal:Cell, volume 167, page 1,144 (issn: 0092-8674,

Authors: Courtney S. Young; April D. Pyle;

doi: 10.1016/j.cell.2016.10.050

pmid: 27863231

Exon Skipping Therapy

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Abstract

Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients.

Related Organizations

University of California, Los Angeles
United States

Keywords

Dystrophin, Muscular Dystrophy, Duchenne, Reading Frames, United States Food and Drug Administration, Humans, Exons, Genetic Therapy, United States

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