title: Exon Skipping Therapy.
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.
authors.affiliation: Molecular Biology Interdepartmental Program, Center for Duchenne Muscular Dystrophy, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095.
authors.forename: Courtney S
authors.initials: CS
authors.lastname: Young
authors.affiliation: Molecular Biology Interdepartmental Program, Center for Duchenne Muscular Dystrophy, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095. Electronic address: apyle@mednet.ucla.edu.
authors.forename: April D
authors.initials: AD
authors.lastname: Pyle
mesh_terms: D016189:Dystrophin / Q000235:genetics*; D005091:Exons; D015316:Genetic Therapy; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy*; D016364:Reading Frames; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D016189:Dystrophin