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Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050.

Exon Skipping Therapy.

Author information

1
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.
2
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.

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.

PMID:
27863231
DOI:
10.1016/j.cell.2016.10.050
[Indexed for MEDLINE]
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