About the Authors

Ian R. Woodcock

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

ian.woodcock@rch.org.au

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Paediatrics, University of Melbourne, Melbourne, Australia

George Tachas

Roles Conceptualization, Funding acquisition, Methodology, Resources, Writing – original draft, Writing – review & editing

Affiliation Antisense Therapeutics Ltd, Melbourne, Australia

Nuket Desem

Roles Formal analysis, Project administration, Writing – review & editing

Affiliation Antisense Therapeutics Ltd, Melbourne, Australia

Peter J. Houweling

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

Affiliations The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Paediatrics, University of Melbourne, Melbourne, Australia

Michael Kean

Roles Data curation, Software, Writing – review & editing

Affiliation Department of Medical Imaging, The Royal Children’s Hospital, Melbourne, Australia

Jaiman Emmanuel

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

Affiliation Department of Medical Imaging, The Royal Children’s Hospital, Melbourne, Australia

Rachel Kennedy

Roles Project administration, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Physiotherapy, University of Melbourne, Melbourne, Australia

Kate Carroll

Roles Project administration, Supervision, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia

Katy de Valle

Roles Project administration, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Physiotherapy, University of Melbourne, Melbourne, Australia

Justine Adams

Roles Writing – review & editing

Affiliation The Murdoch Children’s Research Institute, Melbourne, Australia

Shireen R. Lamandé

Roles Resources, Supervision, Writing – review & editing

Affiliations The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Paediatrics, University of Melbourne, Melbourne, Australia

Chantal Coles

Roles Formal analysis, Writing – review & editing

Affiliation The Murdoch Children’s Research Institute, Melbourne, Australia

Chrystal Tiong

Roles Formal analysis, Methodology, Writing – review & editing

Affiliation The Murdoch Children’s Research Institute, Melbourne, Australia

Matthew Burton

Roles Writing – review & editing

Affiliation The Murdoch Children’s Research Institute, Melbourne, Australia

Daniella Villano

Roles Supervision, Writing – review & editing

Affiliation Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia

Peter Button

Roles Formal analysis, Software, Writing – review & editing

Affiliation McCloud Consulting Group, Sydney, Australia

Jean-Yves Hogrel

Roles Formal analysis, Writing – review & editing

Affiliation Institut de Myologie, GH Pitié-Salpêtrière, Paris, France

Sarah Catling-Seyffer

Roles Project administration, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia

Monique M. Ryan

Roles Conceptualization, Methodology, Supervision, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Paediatrics, University of Melbourne, Melbourne, Australia

Martin B. Delatycki

Roles Supervision, Writing – review & editing

Affiliations Victorian Clinical Genetics Service, Melbourne, Australia, Murdoch Children’s Research Institute, Bruce Lefroy Centre for Genetic Health Research, Melbourne, Australia

Eppie M. Yiu

Roles Supervision, Writing – original draft, Writing – review & editing

Affiliations Department of Neurology, The Royal Children’s Hospital, Melbourne, Australia, The Murdoch Children’s Research Institute, Melbourne, Australia, Department of Paediatrics, University of Melbourne, Melbourne, Australia

Competing Interests

The ATL1102 in DMD clinical trial was funded in its entirety by the commercial sponsor Antisense Therapeutics Ltd. Antisense Therapeutics Ltd is a publicly traded company, listed on the Australian ASX. At the time of the trial, authors Ms Desem and Dr Tachas were employees of the sponsor and so received payment for services from the sponsor as employees. Ms. Desem and Dr Tachas hold an equity interest in the sponsor. Dr Tachas and Ms Desem along with other sponsor employees and sub-contracted specialists were involved in the study design and data analysis. Ms Desem has since left the company and no longer is employed by the sponsor. Authors Dr Woodcock and Dr Ryan were at the time of the trial employees of the Royal Children’s Hospital and Murdoch Children’s Research Institute and are not affiliated with the sponsor in any way and have not received any direct personal payment or honoraria from the sponsors, nor do they or their family members hold a financial interest or stock in the sponsor company. Dr Woodcock is still an employee of the above institutions, but Dr Ryan has since left the employment to take up public office as a Member of the Australian Parliament. Dr Woodcock and Dr Ryan were involved in the trial design as unpaid consultants. Dr Woodcock has received honoraria for work performed including educational activities and attendance at advisory board meetings from pharmaceutical companies Biogen, Novartis, Roche and Avidity and an educational travel bursary to attend an international conference in 2016 from Biogen. Dr Woodcock has received grants for research work from FSHD Global Research Foundation, FSHD Society and Fulcrum Therapeutics. Dr Woodcock has been principal investigator on a number of industry-sponsored clinical trials. None of these disclosures affected the work Dr Woodcock performed on this clinical trial. Dr Ryan has received honoraria for work performed including educational activities and attendance at advisory board meetings from pharmaceutical companies Biogen, Novartis, Roche. Dr Ryan has been principal investigator on a number of industry-sponsored clinical trials. None of these disclosures affected the work Dr Ryan performed on this clinical trial. Dr Yiu has received advisory board honoraria from Biogen and Roche, and has received research support from Biogen, Roche, Pfizer and PTC therapeutics unrelated to the content of this manuscript. Dr Yiu has been principal investigator on a number of industry-sponsored clinical trials. None of these disclosures affected the work Dr Yiu performed on this clinical trial. Prof. Delatycki has received grant awards from NHMRC and is principal investigator in industry sponsored clinical trials including trials sponsored by Rearta and PTC. As this was a clinical trial, publication was always planned from trial inception. No employees of the sponsor were involved in the data collection, although Ms Desem did liaise closely with the MCRI/RCH site staff and Clinical Trial Organisation throughout the trial. As the study statistician, author Dr Button was paid a consultancy fee for his services from the trial sponsor commercial company Antisense Therapeutics Ltd. Authors Dr Houweling, Dr Coles and Dr Tiong were recipients of a grant to perform the MDX studies. This grant was paid by the sponsor Antisense Therapeutics Ltd. None of the other authors received any payment from the sponsor to conduct this study. All other authors had input into writing or revising this manuscript. The authors confirm that the involvement of employees of the sponsor Antisense Therapeutics Ltd in the trial design, data analysis and decision to publish this data does not alter our adherence to PLOS ONE policies on sharing data and materials.