About the Authors

Alper Yavas

Roles Conceptualization, Data curation, Formal analysis, Methodology, Software, Writing – original draft, Writing – review & editing

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands

Rudie Weij

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Software, Supervision, Writing – original draft, Writing – review & editing

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Maaike van Putten

Roles Conceptualization, Data curation, Formal analysis, Methodology, Writing – original draft, Writing – review & editing

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands

Eleni Kourkouta

Roles Data curation, Methodology, Writing – review & editing

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Chantal Beekman

Roles Conceptualization, Data curation, Formal analysis, Methodology, Writing – review & editing

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Jukka Puoliväli

Roles Data curation, Methodology, Writing – review & editing

Affiliation Charles River Discovery Services, Kuopio, Finland

Timo Bragge

Roles Conceptualization, Data curation, Methodology, Writing – review & editing

Affiliation Charles River Discovery Services, Kuopio, Finland

Toni Ahtoniemi

Roles Data curation, Methodology, Writing – review & editing

Affiliation Charles River Discovery Services, Kuopio, Finland

Jeroen Knijnenburg

Roles Methodology, Software, Writing – review & editing

Affiliation Department of Clinical Genetics, Leiden University Medical Center, Leiden, The Netherlands

Marlies Elisabeth Hoogenboom

Roles Methodology

Affiliation Department of Clinical Genetics, Leiden University Medical Center, Leiden, The Netherlands

Yavuz Ariyurek

Roles Investigation, Methodology, Software

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands

Annemieke Aartsma-Rus

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Supervision, Writing – original draft, Writing – review & editing

a.m.rus@lumc.nl

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands

Judith van Deutekom

Roles Conceptualization, Project administration, Supervision, Writing – original draft, Writing – review & editing

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Nicole Datson

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Supervision, Writing – original draft, Writing – review & editing

Affiliation BioMarin Nederland BV, Leiden, The Netherlands

Competing Interests

AAR discloses being employed by LUMC which has patents on exon skipping technology, some of which has been licensed to BioMarin and subsequently sublicensed to Sarepta. As co-inventor of some of these patents AAR is entitled to a share of royalties. AAR further discloses being ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, CRISPR Therapeutics, Summit PLC, Alpha Anomeric, BioMarin Pharmaceuticals Inc., Eisai, Astra Zeneca, Santhera, Audentes, Global Guidepoint and GLG consultancy, Grunenthal, Wave and BioClinica, having been a member of the Duchenne Network Steering Committee (BioMarin) and being a member of the scientific advisory boards of ProQR, Sarepta, Silence and Philae Pharmaceuticals. Remuneration for these activities is paid to LUMC. LUMC also received speaker honoraria from PTC Therapeutics and BioMarin Pharmaceuticals and funding for contract research from Italpharmaco and Alpha Anomeric. RW, EK, CB, JvD and ND are (former) employees of BioMarin Nederland BV (formerly Prosensa Therapeutics BV) and performed the work with company budget in the form of salaries, equipment and facilities. JvD discloses being co-inventor on patents on exon skipping technology, some of which has been licensed to BioMarin and subsequently sublicensed to Sarepta. As co-inventor of some of these patents JvD is entitled to a share of royalties. JP, TB and TA are employees of Charles River Research Discovery Services in Finland and have no financial conflict of interest related to the submitted manuscript. Other authors have nothing to declare. The commercial affiliations of authors with Biomarin Pharmaceutical Ltd. and Charles River do not alter our adherence to PLOS ONE policies on sharing data and materials.