Exon Skipping Therapy

@article{Young2016ExonST,
  title={Exon Skipping Therapy},
  author={Courtney S. Young and April D Pyle},
  journal={Cell},
  year={2016},
  volume={167}
}
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. 
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