Text and Data Mining valid from 2019-03-23
Received: 19 November 2018
Accepted: 25 February 2019
First Online: 23 March 2019
Compliance with ethical standards
: Ethical approval was not obtained since this is a literature review article.
: Q. Ke, Z. Zhao, M. Baker, V. Wiley, J. M. Kwon, L. N. Alfano, C. Jay, E. Ciafaloni, M. Qi, M. A. Gatheridge: no financial benefits have been received or will be received from any party related directly or indirectly to the subject of this article; J. R. Mendell: Dr. Mendell is a Consultant for Sarepta Therapeutics, Avexis (Novartis) therapeutics, and Exonics. He receives consulting fees but no salary. He is reimbursed for presenting clinical trials that pertain to the products sponsored by these companies. He also serves on the Scientific Advisory Boards of the companies. He has no interests from or investments in products that he tests in clinical trials; A. M. Connolly: Dr. Connolly is site principal investigator for multiple studies funded by industry (Sarepta, Avexis, Biogen, PTC Therapeutics, Pfizer, BMS, Fibrogen, Italafarma and NS Pharma). In the last 2 years, Dr. Connolly has served on advisory boards for Sarepta, Avexis, Genetech-Roche, Acceleron, Astelles, Mallincrodt, and SMA Foundation. She is also on the DMSB for Catabasis. Dr. Connolly does not have stock in any company; H. Polari: employee of PerkinElmer Inc; R. C. Griggs: Dr. Griggs has received a research grant support from Marathon, PTC and Sarepta Pharmaceuticals for work related to Duchenne muscular dystrophy. He is also supported by the U.S. National Institutes of Health, the Muscular Dystrophy Association and the Parent Project for Muscular Dystrophy for research on Duchenne muscular dystrophy. He consults for Solid Bioscience.