About the Authors

Ruurd C. Verheul

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Judith C. T. van Deutekom

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Nicole A. Datson

nicole.datson@bmrn.com

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Competing Interests

All authors are employees (which includes contribution to patent [applications] and participation in stock-option plans) of BioMarin Nederland BV, a company that develops RNA therapeutics for Duchenne muscular dystrophy. JCTvD is inventor on exon skipping patents (WO2002/024906 Induction of exon skipping in eukaryotic cells, WO2004/083446 Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure, WO2006/112705 Modulation of exon recognition in premRNA by interfering with the binding of SR proteins and by interfering with secondary RNA structure, WO2009/054725 Means and methods for counteracting muscle disorders, WO2009/139630 Method for efficient exon 44 skipping in Duchenne muscular dystrophy and associated means, WO2010/050801 Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA, WO2010/050802 Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53, WO2013/112053 RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne en Becker muscular dystrophy, WO2014/007620 Oligonucleotide for the treatment of muscular dystrophy patients). This did not alter the authors' adherence to PLOS ONE policies on sharing data and materials.

Author Contributions

Conceptualization: NAD JCTvD. Formal analysis: RCV NAD. Investigation: RCV. Methodology: RCV NAD. Project administration: NAD. Resources: JCTvD. Supervision: NAD JCTvD. Validation: RCV NAD. Visualization: RCV NAD. Writing – original draft: RCV NAD. Writing – review & editing: NAD JCTvD.