About the Authors

Nicole A. Datson

nicole.datson@bmrn.com

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Anchel González-Barriga

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Eleni Kourkouta

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Rudie Weij

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Jeroen van de Giessen

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Susan Mulders

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Outi Kontkanen

Affiliation Charles River Discovery Research Services, Kuopio, Finland

Taneli Heikkinen

Affiliation Charles River Discovery Research Services, Kuopio, Finland

Kimmo Lehtimäki

Affiliation Charles River Discovery Research Services, Kuopio, Finland

Judith C. T. van Deutekom

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Competing Interests

NAD, AG-B, EK, RW, JvdG, SM and JCTvD are (former) employees of BioMarin Nederland BV (formerly Prosensa Therapeutics BV) that sponsored this study. SM is inventor on related patent WO2013/162363. OK, TH and KL are employees of Charles River Research Discovery Services in Finland and have no financial conflict of interest related to the submitted manuscript. This does not alter the authors' adherence to PLOS ONE policies on sharing data and materials.

Author Contributions

Conceptualization: NAD JCTvD OK TH. Formal analysis: JvdG SM AG-B RW EK KL TH NAD. Funding acquisition: NAD JCTvD. Investigation: JvdG SM AG-B RW EK KL TH. Methodology: JvdG SM AG-B RW EK KL TH. Project administration: NAD JCTvD. Supervision: NAD. Validation: JvdG SM AG-B RW EK KL TH. Visualization: EK NAD. Writing – original draft: NAD JCTvD. Writing – review & editing: NAD JCTvD AG-B.