Funding text :
Francesco Muntoni is a member of the Rare Disease Scientific Advisory Group for Pfizer and of Dyne Therapeutics SAB, and has participated to SAB meetings for PTC, Sarepta, Santhera, Wave Therapeutics. UCL and Great Ormond Street Hospital are recipient of grants from Pfizer, Italfarmaco, Wave, Santhera, Sarepta regarding clinical trials. 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 membership of cTAP to conduct this study. Gautam Sajeev, Nicolae Done, Hallee Wong, Jackson Moss, and Zhiwen Yao are employees of Analysis Group, Inc., a consulting firm that received funding from the membership of cTAP to conduct 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. Brenda Wong has participated in advisory committee meetings for Prosensa and Biomarin and has received compensation for consultancy services for Gilead Sciences, Pfizer and GSK. Cuixia Tian has served as the site investigator for trials sponsored by PTC Therapeutics, Eli Lilly, GSK, Prosensa/Biomarin, Bristol Myers Squibb, Roche, Pfizer. Santhera, Sarepta, Fibrogen, Capricor, Pfizer, Avexis, and Catabasis. 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. Susan J. Ward co-founded and manages the collaborative Trajectory Analysis Project and has received funding from the membership of cTAP to facilitate this study. Adnan Manzur has no disclosures. Laurent Servais is member of the SAB or has performed consultancy for Sarepta, Dynacure, Santhera, Avexis, Biogen, Cytokinetics and Roche, Audentes Therapeutics and Affinia Therapeutics; Laurent Servais has given lectures and has served as a consultant for Roche, Biogen, Avexis, and Cytokinetics. Laurent Servais is the project leader of the newborn screening in Southern Belgium funded by Avexis, Roche, and Biogen. Erik H. Niks is a member of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO‐NMD). Erik H. Niks reports grants from Duchenne Parent Project, ZonMW and AFM, consultancies for BioMarin and Summit, and worked as local investigator of clinical trials of BioMarin, GSK, Lilly, Santhera, Givinostat, and Roche outside the submitted work. E.H.N. reports ad hoc consultancies for WAVE, Santhera, Regenxbio, and PTC, and he worked as investigator of clinical trials of Italfarmaco, NS Pharma, Reveragen, Roche, WAVE, and Sarepta outside the submitted work. Volker Straub has participated in advisory boards for Audentes Therapeutics, Biogen, Exonics Therapeutics, Italfarmaco S.p.A., Roche, Sanofi Genzyme, Sarepta Therapeutics, Summit Therapeutics, UCB, and Wave Therapeutics. He has research collaborations with Ultragenyx and Sanofi Genzyme. Imelda JM de Groot has no disclosures. 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.The authors are grateful to patients for participating in the clinical assessments and agreeing to make their data available for research. The authors also thank Eli Lilly for contributing placebo arm data from the tadalafil DMD trial. Gloria DeWalt, PhD, an employee of Analysis Group, Inc., assisted with the development of this manuscript. The authors would like to acknowledge investigators and staff from each of the RWD/NHD sources included in this study. The iMDEX study is funded by the Association Française contre les Myopathies (AFM), while the UK North Star is funded by Muscular Dystrophy UK. Francesco Muntoni is partially supported by the National Institute for Health Research (NIHR) Biomedical Research centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London.This study was conducted within the collaborative Trajectory Analysis Project (cTAP), a pre-competitive coalition of academic clinicians, drug developers, and patient foundations formed in 2015 to overcome the challenges of high variation in clinical trials in DMD. cTAP has received sponsorship from Astellas (Mitobridge), BioMarin Pharmaceutical, Bristol Meyers Squibb, Catabasis, Edgewise Therapeutics, FibroGen, Italfarmaco SpA, Marathon Pharmaceuticals, NS Pharma, Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Shire, Solid Biosciences, Summit Therapeutics, Vertex Pharmaceuticals, Parent Project Muscular Dystrophy, Charley's Fund, and CureDuchenne, a founding patient advocacy partner and provider of initial seed funding to cTAP. Physical function testing at Universitaire Ziekenhuizen Leuven was funded by Fonds Spierzieke Kinderen. The PRO-DMD-01 study was sponsored by BioMarin Pharmaceuticals and data were provided to cTAP by CureDuchenne.Francesco Muntoni is a member of the Rare Disease Scientific Advisory Group for Pfizer and of Dyne Therapeutics SAB, and has participated to SAB meetings for PTC, Sarepta, Santhera, Wave Therapeutics. UCL and Great Ormond Street Hospital are recipient of grants from Pfizer, Italfarmaco, Wave, Santhera, Sarepta regarding clinical trials. 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 membership of cTAP to conduct this study. Gautam Sajeev, Nicolae Done, Hallee Wong, Jackson Moss, and Zhiwen Yao are employees of Analysis Group, Inc., a consulting firm that received funding from the membership of cTAP to conduct 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. Brenda Wong has participated in advisory committee meetings for Prosensa and Biomarin and has received compensation for consultancy services for Gilead Sciences, Pfizer and GSK. Cuixia Tian has served as the site investigator for trials sponsored by PTC Therapeutics, Eli Lilly, GSK, Prosensa/Biomarin, Bristol Myers Squibb, Roche, Pfizer. Santhera, Sarepta, Fibrogen, Capricor, Pfizer, Avexis, and Catabasis. 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. Susan J. Ward co-founded and manages the collaborative Trajectory Analysis Project and has received funding from the membership of cTAP to facilitate this study. Adnan Manzur has no disclosures. Laurent Servais is member of the SAB or has performed consultancy for Sarepta, Dynacure, Santhera, Avexis, Biogen, Cytokinetics and Roche, Audentes Therapeutics and Affinia Therapeutics; Laurent Servais has given lectures and has served as a consultant for Roche, Biogen, Avexis, and Cytokinetics. Laurent Servais is the project leader of the newborn screening in Southern Belgium funded by Avexis, Roche, and Biogen. Erik H. Niks is a member of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO?NMD). Erik H. Niks reports grants from Duchenne Parent Project, ZonMW and AFM, consultancies for BioMarin and Summit, and worked as local investigator of clinical trials of BioMarin, GSK, Lilly, Santhera, Givinostat, and Roche outside the submitted work. E.H.N. reports ad hoc consultancies for WAVE, Santhera, Regenxbio, and PTC, and he worked as investigator of clinical trials of Italfarmaco, NS Pharma, Reveragen, Roche, WAVE, and Sarepta outside the submitted work. Volker Straub has participated in advisory boards for Audentes Therapeutics, Biogen, Exonics Therapeutics, Italfarmaco S.p.A., Roche, Sanofi Genzyme, Sarepta Therapeutics, Summit Therapeutics, UCB, and Wave Therapeutics. He has research collaborations with Ultragenyx and Sanofi Genzyme. Imelda JM de Groot has no disclosures. 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.The authors are grateful to patients for participating in the clinical assessments and agreeing to make their data available for research. The authors also thank Eli Lilly for contributing placebo arm data from the tadalafil DMD trial. Gloria DeWalt, PhD, an employee of Analysis Group, Inc. assisted with the development of this manuscript. The authors would like to acknowledge investigators and staff from each of the RWD/NHD sources included in this study. The iMDEX study is funded by the Association Fran?aise contre les Myopathies (AFM), while the UK North Star is funded by Muscular Dystrophy UK. Francesco Muntoni is partially supported by the National Institute for Health Research (NIHR) Biomedical Research centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. M. Van den Hauwe, N. Goemans, N. Deconicnk, M. Tulinius, E. Niks, I. de Groot, K. Flanigan, E. Henricson, B. Wong, M. Bernadete Dutra de Resende, G. Vita, U. Schara, J. Kirschner, E. Mercuri, H. Topaloglu, D. Monges, M. Soledad, C. Cances, V. Selby, D. Ridout, V. Decostre, A. Mayhew, M. Guglieri, M. Van der Holst, M. Jansen, J.J.G.M. Verschuuren, I.J.M. de Groot, E.H. Niks, L. Servais, V. Straub, J.Y. Hogrel, F. Muntoni, F. Muntoni, A.Y. Manzur, S. Robb, R. Quinlivan, A. Sarkozy, P. Munot, M. Main, L.E. Abbot, H. Patel, S. Samsuddin, V. Ayyar-Gupta, K. Bushby, V. Straub, M. Guglieri, C. Bertolli, A. Mayhew, R. Muni-Lofra, M. James, D. Moat, J. Sodhi, H. Roper, D. Parasuraman, H. McMurchie, R.M. Rabb, A. Childs, K. Pysden, L. Pallant, S. Spinty, G. Peachey, R. Madhu, A.J. Shillington, E. Wraige, H. Jungbluth, V. Gowda, J. Sheehan, R. Spahr, I. Hughes, E. Bateman, C. Cammiss, T. Willis, L. Groves, N. Emery, P. Baxter, M.T. Ong, N. Goulborne, M. Senior, E. Scott, L. Hartley, L.B. Parsons, A. Majumdar, K. Vijaykumar, F.F. Mason, L. Jenkins, B. Toms, C.H. Frimpong-Ansah, K. Naismith, J. Dalgleish, A. Keddie, I. Horrocks, M. Di Marco, J. Dunne, G.C.S. Chow, A. Miah, C. de Goede, A. Selley, N. Thomas, M. Illingworth, M. Geary, J. Palmer, C.P. White, K. Greenfield, S. Tiraputhi, S. MacAuley, N. Hussain, H. Robbins, M. Iqbal, G. Ambegaonkar, D. Krishnakumar, C. Ward, J. Taylor, A. O'Hara, J. Tewnion, S.R. Chandratre, S. Ramdas, M. White, H. Ramjattan, A. Baxter, J. Yirrel, Neuromuscular providers (B. Wong, C. Tian, I. Rybalsky, K. Shellenbarger) and physical therapists (A. McCormick, M. McGuire, K. Bonnarrigo, A. Fowler, M. Kiefer) and research support (J. Bange, S. Hu), E. Mercuri, M. Pane, E. Mazzone, S. Messina, G.L. Vita, M.P. Sormani, A. D'Amico, E. Bertini, A. Berardinelli, F. Magri, G. Pietro Comi, G. Baranello, T. Mongini, A. Pini, R. Battini, E. Pegoraro, C. Bruno, L. Politano, S. Previtali, This study was conducted within the collaborative Trajectory Analysis Project (cTAP), a pre-competitive coalition of academic clinicians, drug developers, and patient foundations formed in 2015 to overcome the challenges of high variation in clinical trials in DMD. cTAP has received sponsorship from Astellas (Mitobridge), BioMarin Pharmaceutical, Bristol Meyers Squibb, Catabasis, Edgewise Therapeutics, FibroGen, Italfarmaco SpA, Marathon Pharmaceuticals, NS Pharma, Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Shire, Solid Biosciences, Summit Therapeutics, Vertex Pharmaceuticals, Parent Project Muscular Dystrophy, Charley's Fund, and CureDuchenne, a founding patient advocacy partner and provider of initial seed funding to cTAP. Physical function testing at Universitaire Ziekenhuizen Leuven was funded by Fonds Spierzieke Kinderen. The PRO-DMD-01 study was sponsored by BioMarin Pharmaceuticals and data were provided to cTAP by CureDuchenne.
