About the Authors

Craig M. McDonald

Roles Conceptualization, Investigation, Resources, Supervision, Writing – original draft, Writing – review & editing

Affiliation Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California Davis Health System, Sacramento, California, United States of America

James Signorovitch

Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Methodology, Project administration, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

james.signorovitch@analysisgroup.com

Affiliations Analysis Group Inc., Boston, Massachusetts, United States of America, Collaborative Trajectory Analysis Project, Cambridge, Massachusetts, United States of America

Eugenio Mercuri

Roles Investigation, Resources, Writing – review & editing

Affiliation Child Neurology Unit e Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Università Cattolica del Sacro Cuore, Rome, Italy

Erik H. Niks

Roles Investigation, Resources, Writing – review & editing

Affiliation Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands

Brenda Wong

Roles Investigation, Resources, Writing – review & editing

Affiliation Department of Pediatrics and Neurology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America

Mirko Fillbrunn

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

Affiliation Analysis Group Inc., Boston, Massachusetts, United States of America

Gautam Sajeev

Roles Data curation, Formal analysis, Project administration, Software, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Analysis Group Inc., Boston, Massachusetts, United States of America

Erica Yim

Roles Data curation, Formal analysis, Software, Validation, Visualization, Writing – review & editing

Affiliation Analysis Group Inc., Boston, Massachusetts, United States of America

Ibrahima Dieye

Roles Data curation, Formal analysis, Software, Validation, Visualization, Writing – review & editing

Affiliation Analysis Group Inc., Boston, Massachusetts, United States of America

Debra Miller

Roles Funding acquisition, Writing – review & editing

Affiliation CureDuchenne, Newport Beach, California, United States of America

Susan J. Ward

Roles Conceptualization, Funding acquisition, Project administration, Supervision, Writing – review & editing

Affiliation Collaborative Trajectory Analysis Project, Cambridge, Massachusetts, United States of America

Nathalie Goemans

Roles Conceptualization, Supervision, Writing – review & editing

Affiliation University Hospitals, Child Neurology, Leuven, Belgium

Investigators from the PRO-DMD-01 Study, Collaborative Trajectory Analysis Project (cTAP)

Membership of the PRO-DMD-01 is provided in the Acknowledgements. Membership of the Collaborative Trajectory Analysis project can be found at https://ctap-duchenne.org/our-team/.

Membership of the PRO-DMD-01 is provided in the Acknowledgements. Membership of the Collaborative Trajectory Analysis project can be found at https://ctap-duchenne.org/our-team/.

Competing Interests

Craig McDonald has served as a consultant for PTC Therapeutics, BioMarin Pharmaceutical, Sarepta Therapeutics, Eli Lilly, Pfizer Inc, Santhera Pharmaceuticals, Cardero Therapeutics, Inc, Catabasis Pharmaceuticals, Capricor Therapeutics, Astellas Pharma (Mitobridge), and FibroGen, Inc; serves on external advisory boards related to DMD for PTC Therapeutics, Sarepta Therapeutics, Santhera Pharmaceuticals, and Capricor Therapeutics; and reports grants from US Department of Education/National Institute on Disability and Rehabilitation Research, the National Institute on Disability, Independent Living, and Rehabilitation Research, US NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH/National Institute of Neurologic Disorders and Stroke, US Department of Defense, and Parent Project Muscular Dystrophy US. James Signorovitch co-founded the collaborative Trajectory Analysis Project (cTAP) and is an employee of Analysis Group, Inc., a consulting firm that received funding from the sponsors of cTAP to conduct this study. Eugenio Mercuri has served on clinical steering committees and/or as a consultant for Eli Lilly, Italfarmaco, PTC Therapeutics, Sarepta, Santhera, and Pfizer; has served as PI for GlaxoSmithKline, Prosensa, BioMarin Pharmaceutical, Italfarmaco, Roche, PTC, Pfizer, Sarepta, Santhera, Wave, NS and Eli Lilly. Erik H. Niks is a member of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO‐NMD). Erik H. Niks report grants from Spieren voor Spieren, Duchenne Parent Project, ZonMW, AFM and PPMD.He has been site principal investigator for clinical trials conducted by BioMarin, GSK, Eli Lilly, Santhera Pharmaceuticals, Italfarmaco SpA, Roche Pharma, Reveragen, NS Pharma, Fibrogen, Sarepta, Alexion, Janssen and Argnx outside the submitted work. He also reports ad hoc consultancies for BioMarin, Summit, PTC therapeutics, WAVE Life Sciences, Edgewise, Epirium Bio, Janssen, Sarepta and Regenxbio. All reimbursements were received by the LUMC. No personal financial benefits were received. Brenda Wong has participated in advisory committee meetings for Prosensa and Biomarin and has received compensation for consultancy services for Gilead Sciences, Pfizer, GSK and PepGen. Mirko Fillbrunn is an employee of Analysis Group, Inc., a consulting firm that received funding from the sponsors of cTAP to conduct this study. Gautam Sajeev is an employee of Analysis Group, Inc., a consulting firm that received funding from the sponsors of cTAP to conduct this study. Erica Yim was an employee of Analysis Group, Inc., a consulting firm that received funding from the sponsors of cTAP to conduct this study. Ibrahima Dieye was an employee of Analysis Group, Inc., a consulting firm that received funding from the sponsors of cTAP to conduct this study. Susan J. Ward co-founded and manages the collaborative Trajectory Analysis Project and has received funding from the sponsors of cTAP to facilitate this study. Nathalie Goemans has received compensation for consultancy services from Eli Lilly, Italfarmaco, PTC Therapeutics, BioMarin Pharmaceutical, Pfizer, Avidity, Daiichi Sankyo, Wave, Santhera and has served as site investigator for GlaxoSmithKline, Prosensa, BioMarin Pharmaceutical, Italfarmaco, Eli Lilly, Wave, and Sarepta. This does not alter our adherence to PLOS ONE policies on sharing data and materials.