Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.

"Conflicts of Interest: Dr. Darras has served as an ad hoc scientific advisory board member for AveXis, Biogen, Cytokinetics, Marathon Pharmaceuticals, PTC Therapeutics, Bristol‐Myers Squibb, Roche, and Sarepta; and has been an advisor for Ionis Pharmaceuticals, Inc.; he has no financial interests in these companies. He has received research support from the National Institutes of Health/National Institute of Neurological Disorders and Stroke, the Slaney Family Fund for SMA, the Spinal Muscular Atrophy Foundation, CureSMA, and Working on Walking Fund; and grants from Ionis Pharmaceuticals, Inc. for the ENDEAR, CHERISH, CS2/CS12 studies, from Biogen for CS11, as well as from Cytokinetics, Sarepta Pharmaceuticals, PTC Therapeutics, Fibrogen, and Summit. Dr. Shieh has served as an ad hoc advisory board member for AveXis, Biogen, Marathon, PTC, and Sarepta but he has no financial interests in these companies. He has received research support from Ionis Pharmaceuticals and Biogen for their SMA studies, as well as from Cytokinetics for their SMA clinical trial, Sarepta for the DMD clinical trials, PTC Therapeutics for their DMD/ataluren trial, Pfizer for their DMD/myostatin clinical trial, BMS/Roche for their DMD/myostatin clinical trial, Sanofi/Genzyme for their Pompe clinical trial, and Summit for their DMD clinical trial. Dr. McDonald has served as a consultant for clinical trials for PTC Therapeutics, Biomarin, Sarepta, Eli Lilly, Pfizer, Santhera Pharmaceuticals, Cardero Therapeutics, Inc., Catabasis, Capricor, Marathon, and Mitobridge, outside the submitted work; serves on external advisory boards related to Duchenne muscular dystrophy for PTC Therapeutics, Eli Lilly, Sarepta Therapeutics, Santhera Pharmaceuticals, and Capricor; and reports grants from US Dept. of Education/NIDRR, NIDILRR, US NIH/NIAMS, US Department of Defense, and Parent Project Muscular Dystrophy US, during the conduct of the study. Joseph McIntosh, Fengbin Jin, Gary Elfring, Marcio Souza, Siva Narayanan, Panayiota Trifillis, and Stuart W. Peltz are employees and stockholders of PTC Therapeutics, Inc."

"We thank the patients and their families for their participation in this study; individuals who were instrumental in the conduct of this study and the collection of data, particularly principal investigators, supporting investigators, clinical coordinators, clinical evaluators, and study coordinators. We thank Robert Weiss (University of Utah, UT, USA) for dystrophin gene sequencing; the patient advocacy organizations (including Valerie Cwik and the Muscular Dystrophy Association, and Patricia Furlong and the Parent Project Muscular Dystrophy) for the collaboration and support that made this trial possible. Medical writing was provided by Annette Skorupa, PhD of EnlightenMed LLC and was funded by PTC Therapeutics, Inc. The ACT DMD Study Group: Hoda Z Abdel‐Hamid (Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA); Susan D Apkon (Seattle Children's Hospital, Seattle, WA, USA); Richard J Barohn (University of Kansas Medical Center, Kansas City, KS, USA); Enrico Bertini (Bambino Gesù Children's Research Hospital, Rome, Italy); Clemens Bloetzer (Unité de Neurologie et Neuroréhabilitation Pédiatrique, Département de Pédiatrie, CHUV, Lausanne Canton de Vaud, Switzerland); Russell J Butterfield (University of Utah, Department of Neurology and Pediatrics, Salt Lake City, UT, USA); Craig Campbell (Schulich School of Medicine and Dentistry, Western University, Children's Health Research Institute, London, ON, Canada), Brigitte Chabrol (Hôpital de la Timone, Unité de Médecine Infantile, Marseille, France); Jong‐Hee Chae (Seoul National University Children's Hospital, 101 Daehak‐ro Jongno‐gu, Seoul, South Korea); Giacomi Pietro Comi (IRCCS Foundation Ca'Granda, Ospedale Maggiore Policlinico, Dino Ferrari Centre, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy); Basil T Darras (Boston Children's Hospital, Harvard Medical School, Boston, MA, USA); Jahannaz Dastgir (Goryeb Children's Hospital, Morristown, NJ, USA); Isabelle Desguerre (Necker‐Enfants Malades, Bâtiment Robert Debré, Paris, France); Raul G Escobar (Neurology Unit, Division of Pediatrics, Medical School, Pontificia Universidad Catolica de Chile, Santiago, Chile); Erika Finanger (Oregon Health and Science University, Portland, OR, USA); Richard S Finkel (Children's Hospital of Philadelphia, Philadelphia, PA, USA; Nemours Children's Hospital, Orlando, FL, USA); Kevin M Flanigan (Nationwide Children's Hospital, Columbus, OH, USA); Nathalie Goemans (University Hospitals Leuven, KU Leuven, Belgium); Michela Guglieri (Institute of Genetic Medicine International Centre for Life, Central Parkway, Newcastle‐upon‐Tyne, UK); Peter Heydemann (Rush University Medical Center, Chicago, IL, USA); Imelda Hughes (Royal Manchester Children's Hospital, Oxford Road, Manchester, UK); Susan T Iannaccone (University of Texas Southwestern Medical Center, Dallas, TX, USA); Kristi J Jones (Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead, Westmead, NSW, Australia); Anna Kaminska (Medical University of Warsaw, Warsaw, Poland); Peter Karachunski (University of Minnesota, Department of Neurology, Minneapolis, MN, USA); Janbernd Kirschner (Medical Center ‐ University of Freiburg, University of Freiberg, Freiberg, Germany); Martin Kudr (Department of Paediatric Neurology, Charles University, 2nd Faculty of Medicine, Motol University Hospital, Prague, Czech Republic); Timothy Lotze (Texas Children's Hospital, Houston, TX, USA); Jean K Mah (Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada); Katherine Mathews (Department of Pediatrics, Division of Pediatric Neurology, University of Iowa Children's Hospital, Iowa City, IA, USA); Craig M McDonald (University of California at Davis Medical Center, Sacramento, CA, USA); Eugenio Mercuri (Department of Pediatric Neurology, Catholic University, Rome, Italy); Francesco Muntoni (University College London Great Ormond Street Institute of Child Health, London, UK); Yoram Nevo (Hadassah Medical Center, Mount Scopus, Jerusalem, Israel); Andrés Nascimento Osorio (Hospital Sant Joan de Déu, Universidad de Barcelona, CIBER, ISCIII, Barcelona, Spain); Julie Parsons (Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA); Yann Péréon (Reference Centre for Neuromuscular Disorders, CHU Nantes, Nantes, France); Alexandra Prufer de Queiroz Campos Araujo (Rua Bruno Lobo, 50‐3º andar–Departamento de Pediatria, Cidade Universitária–Ilha do Fundão, Rio de Janeiro, Brazil); J Ben Renfroe (Child Neurology Center of Northwest Florida, Gulf Breeze, FL, USA); Maria Bernadete Dutra de Resende (Departamento de Neurologia Infantil, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil); Monique Ryan (The Royal Children's Hospital, Parkville, VIC, Australia); Jacinda Sampson (Stanford University School of Medicine, Stanford, CA, USA); Ulrike Schara (University Hospital Essen, University of Duisburg‐Essen, Essen, Germany); Kathryn Selby (Division of Neurology, British Columbia's Children's Hospital, Vancouver, BC, Canada); Thomas Sejersen (Karolinska University Hospital, Karolinska Institutet, Stockholm, Sweden); Perry B Shieh (University of California at Los Angeles, Los Angeles, CA, USA); H Lee Sweeney (University of Florida, Gainesville, FL, USA); Gihan Tennekoon (The Children's Hospital of Philadelphia, Division of Neurology, Philadelphia, PA, USA); Haluk Topaloglu (Hacettepe Children's Hospital, Ankara, Turkey); Ricardo Erazo Torricelli (Hospital Luis Calvo Mackenna, Santiago de Chile, Chile); Már Tulinius (Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden); Juan J Vilchez (Hospital Universitario y Politécnico La Fe, CIBERER, Valencia, Spain); Giuseppe Vita (University of Messina, AOU Policlinico G Martino, Nemo Sud Clinical Centre, Messina, Italy); Thomas Voit (National Institute for Health Research, Great Ormond Street Hospital, University College London Biomedical Research Centre, London, UK); Brenda Wong (Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA). The Clinical Evaluator Training Group: Lindsay N Alfano (Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA); Michelle Eagle (Atom International, Gateshead, UK); Meredith K James (The John Walton Muscular Dystrophy Research Centre, Newcastle, UK); Linda Lowes (Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA); Anna Mayhew (MRC Centre for Neuromuscular Diseases, Newcastle University, Newcastle, UK); Elena S Mazzone (Department of Pediatric Neurology, Catholic University, Rome, Italy); Leslie Nelson (University of Texas Southwestern Medical Center, TX, USA); Kristy J Rose (Gait Analysis Laboratory of New South Wales, The Children's Hospital at Westmead, Sydney, NSW, Australia). Ethical Publication Statement: We confirm that we have read the Journal's position on issues involved in ethical publication and affirm that this report is consistent with those guidelines."