{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T04:15:15Z","timestamp":1791951315713,"version":"4.3.4"},"reference-count":203,"publisher":"Public Library of Science (PLoS)","issue":"3","license":[{"start":{"date-parts":[[2022,3,25]],"date-time":"2022-03-25T00:00:00Z","timestamp":1648166400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013223","name":"PTC Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013223","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:sec id=\"sec001\">\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Duchenne muscular dystrophy (DMD) is a rare, severely debilitating, and fatal neuromuscular disease characterized by progressive muscle degeneration. Like in many orphan diseases, randomized controlled trials are uncommon in DMD, resulting in the need to indirectly compare treatment effects, for example by pooling individual patient-level data from multiple sources. However, to derive reliable estimates, it is necessary to ensure that the samples considered are comparable with respect to factors significantly affecting the clinical progression of the disease. To help inform such analyses, the objective of this study was to review and synthesise published evidence of prognostic indicators of disease progression in DMD. We searched MEDLINE (via Ovid), Embase (via Ovid) and the Cochrane Library (via Wiley) for records published from inception up until April 23 2021, reporting evidence of prognostic indicators of disease progression in DMD. Risk of bias was established with the grading system of the Centre for Evidence-Based Medicine (CEBM).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"sec002\">\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Our search included 135 studies involving 25,610 patients from 18 countries across six continents (Africa, Asia, Australia, Europe, North America and South America). We identified a total of 23 prognostic indicators of disease progression in DMD, namely age at diagnosis, age at onset of symptoms, ataluren treatment, ATL1102, BMI, cardiac medication, DMD genetic modifiers, DMD mutation type, drisapersen, edasalonexent, eteplirsen, glucocorticoid exposure, height, idebenone, lower limb surgery, orthoses, oxandrolone, spinal surgery, TAS-205, vamorolone, vitlolarsen, ventilation support, and weight. Of these, cardiac medication, DMD genetic modifiers, DMD mutation type, and glucocorticoid exposure were designated core prognostic indicators, each supported by a high level of evidence and significantly affecting a wide range of clinical outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"sec003\">\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This study provides a current summary of prognostic indicators of disease progression in DMD, which will help inform the design of comparative analyses and future data collection initiatives in this patient population.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1371\/journal.pone.0265879","type":"journal-article","created":{"date-parts":[[2022,3,25]],"date-time":"2022-03-25T13:43:10Z","timestamp":1648215790000},"page":"e0265879","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":30,"title":["Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis"],"prefix":"10.1371","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9631-0470","authenticated-orcid":true,"given":"Nermina","family":"Ferizovic","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Summers","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Igor Beitia Ortiz","family":"de Z\u00e1rate","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Werner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik","family":"Landfeldt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katharina","family":"Buesch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2022,3,25]]},"reference":[{"issue":"9307","key":"pone.0265879.ref001","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1016\/S0140-6736(02)07815-7","article-title":"The muscular dystrophies","volume":"359","author":"AE Emery","year":"2002","journal-title":"Lancet"},{"issue":"8","key":"pone.0265879.ref002","doi-asserted-by":"crossref","first-page":"2303","DOI":"10.1093\/nar\/11.8.2303","article-title":"Linkage analysis of two cloned DNA sequences flanking the Duchenne muscular dystrophy locus on the short arm of the human X chromosome","volume":"11","author":"KE Davies","year":"1983","journal-title":"Nucleic Acids Res"},{"issue":"3","key":"pone.0265879.ref003","first-page":"1223","article-title":"The Dystrophin Complex: Structure, Function, and Implications for Therapy","volume":"5","author":"QQ Gao","year":"2015","journal-title":"Compr Physiol"},{"issue":"12","key":"pone.0265879.ref004","doi-asserted-by":"crossref","first-page":"1657","DOI":"10.1002\/humu.21114","article-title":"Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort","volume":"30","author":"KM Flanigan","year":"2009","journal-title":"Hum Mutat"},{"issue":"10","key":"pone.0265879.ref005","doi-asserted-by":"crossref","first-page":"1049","DOI":"10.1038\/ejhg.2012.301","article-title":"Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK)","volume":"21","author":"SJ Moat","year":"2013","journal-title":"Eur J Hum Genet"},{"key":"pone.0265879.ref006","author":"Parent Project Muscular Dystrophy","journal-title":"About Duchenne"},{"issue":"12","key":"pone.0265879.ref007","doi-asserted-by":"crossref","first-page":"1074","DOI":"10.1136\/archdischild-2014-306366","article-title":"Improving recognition of Duchenne muscular dystrophy: a retrospective case note review","volume":"99","author":"HJA van Ruiten","year":"2014","journal-title":"Archives of Disease in Childhood"},{"issue":"10","key":"pone.0265879.ref008","doi-asserted-by":"crossref","first-page":"1294","DOI":"10.1038\/ejhg.2014.301","article-title":"A mixed methods study of age at diagnosis and diagnostic odyssey for Duchenne muscular dystrophy","volume":"23","author":"SH Wong","year":"2015","journal-title":"European journal of human genetics"},{"issue":"2","key":"pone.0265879.ref009","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1152\/physrev.00028.2001","article-title":"Function and Genetics of Dystrophin and Dystrophin-Related Proteins in Muscle","volume":"82","author":"DJ Blake","year":"2002","journal-title":"Physiological Reviews"},{"key":"pone.0265879.ref010","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1186\/s13023-017-0631-3","article-title":"The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review","volume":"12","author":"S Ryder","year":"2017","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"4","key":"pone.0265879.ref011","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1212\/WNL.0000000000002891","article-title":"DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study","volume":"87","author":"L Bello","year":"2016","journal-title":"Neurology"},{"issue":"7","key":"pone.0265879.ref012","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1007\/s10654-020-00613-8","article-title":"Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis","volume":"35","author":"E Landfeldt","year":"2020","journal-title":"Eur J Epidemiol"},{"issue":"4","key":"pone.0265879.ref013","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1016\/S1474-4422(18)30025-5","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management","volume":"17","author":"DJ Birnkrant","year":"2018","journal-title":"Lancet Neurol"},{"issue":"12","key":"pone.0265879.ref014","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1093\/aje\/kwj149","article-title":"Variable selection for propensity score models","volume":"163","author":"MA Brookhart","year":"2006","journal-title":"Am J Epidemiol"},{"key":"pone.0265879.ref015","doi-asserted-by":"crossref","first-page":"b2700","DOI":"10.1136\/bmj.b2700","article-title":"The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate healthcare interventions: explanation and elaboration","volume":"339","author":"A Liberati","year":"2009","journal-title":"BMJ"},{"key":"pone.0265879.ref016","author":"OCEBM Levels of Evidence Working Group","year":"2021","journal-title":"The Oxford Levels of Evidence 2"},{"issue":"4","key":"pone.0265879.ref017","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/j.nmd.2006.01.010","article-title":"Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade","volume":"16","author":"WD Biggar","year":"2006","journal-title":"Neuromuscul Disord"},{"issue":"3","key":"pone.0265879.ref018","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1016\/j.pediatrneurol.2007.11.001","article-title":"Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up","volume":"38","author":"S Houde","year":"2008","journal-title":"Pediatr Neurol"},{"issue":"6","key":"pone.0265879.ref019","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1016\/S0002-9149(02)03429-X","article-title":"Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy","volume":"91","author":"CK Silversides","year":"2003","journal-title":"Am J Cardiol"},{"issue":"4","key":"pone.0265879.ref020","doi-asserted-by":"crossref","first-page":"1080","DOI":"10.1016\/j.jpeds.2013.05.060","article-title":"Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy","volume":"163","author":"BJ Barber","year":"2013","journal-title":"The Journal of Pediatrics"},{"issue":"Suppl 1","key":"pone.0265879.ref021","first-page":"S145","article-title":"Angelo G, Bruno C, Berardinelli A, Comi G, D","volume":"29","author":"D Bello L","year":"2019","journal-title":"Neuromuscular Disorders"},{"issue":"2","key":"pone.0265879.ref022","first-page":"103","article-title":"Modifiers of respiratory and cardiac function in the Italian Duchenne muscular dystrophy Network and CINRG Duchenne Natural History Study","volume":"38","author":"L Bello","year":"2019","journal-title":"Acta myologica Proceedings Of The XIX Congress Of The Italian Society of Myology: Bergamo, Italy"},{"issue":"4","key":"pone.0265879.ref023","doi-asserted-by":"crossref","first-page":"e001338","DOI":"10.1161\/JAHA.114.001338","article-title":"Myocardial fibrosis burden predicts left ventricular ejection fraction and is associated with age and steroid treatment duration in duchenne muscular dystrophy","volume":"4","author":"A Tandon","year":"2015","journal-title":"J Am Heart Assoc"},{"key":"pone.0265879.ref024","first-page":"1819","article-title":"Early myocardial damage assessment in dystrophinopathies using (99)Tc(m)-MIBI gated myocardial perfusion imaging","volume":"11","author":"L Zhang","year":"2015","journal-title":"Ther Clin Risk Manag"},{"issue":"9","key":"pone.0265879.ref025","doi-asserted-by":"crossref","first-page":"948","DOI":"10.1016\/j.jacc.2012.12.008","article-title":"All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy","volume":"61","author":"G Schram","year":"2013","journal-title":"J Am Coll Cardiol"},{"issue":"5","key":"pone.0265879.ref026","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1016\/j.nmd.2008.03.002","article-title":"Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy","volume":"18","author":"LW Markham","year":"2008","journal-title":"Neuromuscul Disord"},{"issue":"6","key":"pone.0265879.ref027","doi-asserted-by":"crossref","first-page":"768","DOI":"10.1007\/s00246-005-0909-4","article-title":"Steroid therapy and cardiac function in Duchenne muscular dystrophy","volume":"26","author":"LW Markham","year":"2005","journal-title":"Pediatr Cardiol"},{"issue":"8","key":"pone.0265879.ref028","doi-asserted-by":"crossref","first-page":"730","DOI":"10.1016\/j.nmd.2017.05.019","article-title":"Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy","volume":"27","author":"S Kim","year":"2017","journal-title":"Neuromuscul Disord"},{"issue":"2","key":"pone.0265879.ref029","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1007\/s00246-018-2046-x","article-title":"Progressive left ventricular dysfunction and myocardial fibrosis in Duchenne and Becker muscular dystrophy: a longitudinal cardiovascular magnetic resonance study","volume":"40","author":"T Aikawa","year":"2019","journal-title":"Pediatr Cardiol"},{"issue":"3","key":"pone.0265879.ref030","doi-asserted-by":"crossref","first-page":"184","DOI":"10.4070\/kcj.2012.42.3.184","article-title":"The effect of enalapril and carvedilol on left ventricular dysfunction in middle childhood and adolescent patients with muscular dystrophy","volume":"42","author":"HW Kwon","year":"2012","journal-title":"Korean Circ J"},{"issue":"8","key":"pone.0265879.ref031","doi-asserted-by":"crossref","first-page":"991","DOI":"10.1253\/circj.70.991","article-title":"Beta-blocker therapy for cardiac dysfunction in patients with muscular dystrophy","volume":"70","author":"H Kajimoto","year":"2006","journal-title":"Circ J"},{"issue":"Suppl 13","key":"pone.0265879.ref032","doi-asserted-by":"crossref","first-page":"E820","DOI":"10.1016\/S0735-1097(12)60821-3","article-title":"Natural history of cardiomyopathy in duchenne muscular dystrophy and the effects of angiotensin-converting enzyme inhibitor with or without beta-blocker","volume":"59","author":"P Thrush","year":"2012","journal-title":"Journal of the American College of Cardiology. Journal of the American College of Cardiology"},{"issue":"Suppl 1","key":"pone.0265879.ref033","first-page":"S15 003","article-title":"Natural history of cardiomyopathy in Duchenne muscular dystrophy and the effects of angiotensin-converting enzyme inhibitor with or without \u03b2-blocker (S15.003)","volume":"78","author":"P Thrush","year":"2012","journal-title":"Neurology"},{"issue":"1","key":"pone.0265879.ref034","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1016\/j.amjcard.2012.02.064","article-title":"Effects of angiotensin-converting enzyme inhibitors and\/or beta blockers on the cardiomyopathy in Duchenne muscular dystrophy","volume":"110","author":"L Viollet","year":"2012","journal-title":"Am J Cardiol"},{"issue":"18","key":"pone.0265879.ref035","doi-asserted-by":"crossref","first-page":"2799","DOI":"10.1161\/CIRCULATIONAHA.104.528281","article-title":"Genetic Predictors and Remodeling of Dilated Cardiomyopathy in Muscular Dystrophy","volume":"112","author":"JL Jefferies","year":"2005","journal-title":"Circulation"},{"issue":"2","key":"pone.0265879.ref036","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1016\/S1474-4422(14)70318-7","article-title":"Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial","volume":"14","author":"SV Raman","year":"2015","journal-title":"Lancet Neurol"},{"issue":"14","key":"pone.0265879.ref037","doi-asserted-by":"crossref","first-page":"1357","DOI":"10.2169\/internalmedicine.49.3259","article-title":"Carvedilol can prevent cardiac events in Duchenne muscular dystrophy","volume":"49","author":"T Matsumura","year":"2010","journal-title":"Intern Med"},{"issue":"Suppl 1","key":"pone.0265879.ref038","doi-asserted-by":"crossref","first-page":"e235","DOI":"10.1016\/j.ejpn.2017.04.1263","article-title":"Impact of three decades of improvements in standards of care on clinical outcomes in Duchenne muscular dystrophy","volume":"21","author":"HJA van Ruiten","year":"2017","journal-title":"European Journal of Paediatric Neurology"},{"issue":"27","key":"pone.0265879.ref039","doi-asserted-by":"crossref","first-page":"e11381","DOI":"10.1097\/MD.0000000000011381","article-title":"Natural history of cardiac function in Duchenne and Becker muscular dystrophies on home mechanical ventilation","volume":"97","author":"A Fayssoil","year":"2018","journal-title":"Medicine (Baltimore)"},{"issue":"10","key":"pone.0265879.ref040","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1016\/j.cardfail.2020.08.002","article-title":"The ACTN3 577XX null genotype is associated with low left ventricular dilation-free survival rate in patients with Duchenne muscular dystrophy","volume":"26","author":"M Nagai","year":"2020","journal-title":"Journal of Cardiac Failure"},{"issue":"10","key":"pone.0265879.ref041","doi-asserted-by":"crossref","first-page":"e006340","DOI":"10.1161\/JAHA.117.006340","article-title":"Predictors of death in adults with Duchenne muscular dystrophy\u2013associated cardiomyopathy","volume":"6","author":"D Cheeran","year":"2017","journal-title":"Journal of the American Heart Association"},{"issue":"6","key":"pone.0265879.ref042","doi-asserted-by":"crossref","first-page":"855","DOI":"10.1016\/j.jacc.2004.09.078","article-title":"Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy","volume":"45","author":"D Duboc","year":"2005","journal-title":"J Am Coll Cardiol"},{"issue":"3","key":"pone.0265879.ref043","doi-asserted-by":"crossref","first-page":"596","DOI":"10.1016\/j.ahj.2007.05.014","article-title":"Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years\u2019 follow-up","volume":"154","author":"D Duboc","year":"2007","journal-title":"Am Heart J"},{"issue":"5","key":"pone.0265879.ref044","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1016\/S0002-8703(99)70414-X","article-title":"Cardioprotection for Duchenne\u2019s muscular dystrophy","volume":"137","author":"Y Ishikawa","year":"1999","journal-title":"American Heart Journal"},{"issue":"6","key":"pone.0265879.ref045","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1016\/j.amjcard.2006.04.020","article-title":"Left ventricular function and response to enalapril in patients with duchenne muscular dystrophy during the second decade of life","volume":"98","author":"C Ramaciotti","year":"2006","journal-title":"Am J Cardiol"},{"issue":"19","key":"pone.0265879.ref046","doi-asserted-by":"crossref","first-page":"1607","DOI":"10.1212\/01.wnl.0000260974.41514.83","article-title":"Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy","volume":"68","author":"WM King","year":"2007","journal-title":"Neurology"},{"issue":"11","key":"pone.0265879.ref047","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1097\/01.phm.0000184156.98671.d0","article-title":"Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect","volume":"84","author":"B Balaban","year":"2005","journal-title":"Am J Phys Med Rehabil"},{"issue":"3","key":"pone.0265879.ref048","doi-asserted-by":"crossref","first-page":"519","DOI":"10.2106\/00004623-200403000-00009","article-title":"Steroid Treatment and the Development of Scoliosis in Males with Duchenne Muscular Dystrophy","volume":"86","author":"BA Alman","year":"2004","journal-title":"J Bone Joint Surg Am"},{"issue":"12","key":"pone.0265879.ref049","doi-asserted-by":"crossref","first-page":"1057","DOI":"10.2106\/JBJS.L.01577","article-title":"Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up","volume":"95","author":"DE Lebel","year":"2013","journal-title":"J Bone Joint Surg Am"},{"issue":"3","key":"pone.0265879.ref050","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1016\/j.ejpn.2006.12.002","article-title":"Predictive factors for the development of scoliosis in Duchenne muscular dystrophy","volume":"11","author":"M Kinali","year":"2007","journal-title":"Eur J Paediatr Neurol"},{"issue":"10119","key":"pone.0265879.ref051","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/S0140-6736(17)32160-8","article-title":"Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study","volume":"391","author":"CM McDonald","year":"2018","journal-title":"The Lancet"},{"issue":"1","key":"pone.0265879.ref052","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1016\/j.jjcc.2008.08.013","article-title":"Beneficial effects of beta-blockers and angiotensin-converting enzyme inhibitors in Duchenne muscular dystrophy","volume":"53","author":"H Ogata","year":"2009","journal-title":"Journal of Cardiology"},{"issue":"2","key":"pone.0265879.ref053","first-page":"117","article-title":"Survival in Duchenne muscular dystrophy","volume":"31","author":"S Rall","year":"2012","journal-title":"Acta Myol"},{"issue":"10","key":"pone.0265879.ref054","doi-asserted-by":"crossref","first-page":"804","DOI":"10.1016\/S0960-8966(03)00162-7","article-title":"The Duchenne muscular dystrophy population in Denmark, 1977\u20132001: prevalence, incidence and survival in relation to the introduction of ventilator use","volume":"13","author":"J Jeppesen","year":"2003","journal-title":"Neuromuscular Disorders"},{"issue":"6","key":"pone.0265879.ref055","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1016\/j.nmd.2007.03.002","article-title":"Managing Duchenne muscular dystrophy\u2013The additive effect of spinal surgery and home nocturnal ventilation in improving survival","volume":"17","author":"M Eagle","year":"2007","journal-title":"Neuromuscular Disorders"},{"issue":"10","key":"pone.0265879.ref056","doi-asserted-by":"crossref","first-page":"926","DOI":"10.1016\/S0960-8966(02)00140-2","article-title":"Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation","volume":"12","author":"M Eagle","year":"2002","journal-title":"Neuromuscul Disord"},{"issue":"6","key":"pone.0265879.ref057","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1097\/00002060-200206000-00003","article-title":"Duchenne muscular dystrophy: prolongation of life by noninvasive ventilation and mechanically assisted coughing","volume":"81","author":"E Gomez-Merino","year":"2002","journal-title":"Am J Phys Med Rehabil"},{"issue":"6","key":"pone.0265879.ref058","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.rehab.2013.06.002","article-title":"Evolution of life expectancy of patients with Duchenne muscular dystrophy at AFM Yolaine de Kepper centre between 1981 and 2011","volume":"56","author":"P Kieny","year":"2013","journal-title":"Annals of physical and rehabilitation medicine"},{"issue":"1","key":"pone.0265879.ref059","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.nmd.2010.09.006","article-title":"Duchenne muscular dystrophy: survival by cardio-respiratory interventions","volume":"21","author":"Y Ishikawa","year":"2011","journal-title":"Neuromuscular Disorders"},{"issue":"Supplement_1","key":"pone.0265879.ref060","doi-asserted-by":"crossref","DOI":"10.1093\/eurheartj\/ehz745.0319","article-title":"P3446Left-ventricular assist device as a destination therapy in Duchenne cardiomyopathy: are we ready to change the natural history?","volume":"40","author":"R Adorisio","year":"2019","journal-title":"European Heart Journal"},{"issue":"9","key":"pone.0265879.ref061","doi-asserted-by":"crossref","first-page":"835","DOI":"10.1016\/j.nmd.2012.06.111","article-title":"G.P.77 Deletions in the dystrophin gene predict loss of ambulation before 10years of age in boys with Duchenne muscular dystrophy","volume":"22","author":"ZE Davidson","year":"2012","journal-title":"Neuromuscular Disorders"},{"issue":"10","key":"pone.0265879.ref062","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1136\/jnnp.2005.078345","article-title":"The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy","volume":"77","author":"DM Bonifati","year":"2006","journal-title":"Journal of neurology, neurosurgery, and psychiatry"},{"issue":"Suppl 14","key":"pone.0265879.ref063","first-page":"S50","article-title":"Prednisone\/prednisolone and deflazacort differ in long term outcomes on ambulation and side effects in the CINRG Duchenne Natural History Study (S50.001)","volume":"84","author":"L Bello","year":"2015","journal-title":"Neurology"},{"issue":"12","key":"pone.0265879.ref064","doi-asserted-by":"crossref","first-page":"1048","DOI":"10.1212\/WNL.0000000000001950","article-title":"Prednisone\/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study","volume":"85","author":"L Bello","year":"2015","journal-title":"Neurology"},{"issue":"4","key":"pone.0265879.ref065","doi-asserted-by":"crossref","first-page":"684","DOI":"10.1002\/ana.24370","article-title":"Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study","volume":"77","author":"L Bello","year":"2015","journal-title":"Ann Neurol"},{"issue":"Suppl 1","key":"pone.0265879.ref066","first-page":"S124","article-title":"Loss of ambulation in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Muscular Dystrophy (DMD) cohort is synergistically infiuenced by glucocorticoid corticosteroid treatment and candidate genetic polymorphisms","volume":"1","author":"L Bello","year":"2014","journal-title":"Journal of Neuromuscular Diseases 13th International congress on Neuromuscular Diseases"},{"key":"pone.0265879.ref067","doi-asserted-by":"crossref","first-page":"S119","DOI":"10.1016\/j.nmd.2016.06.124","article-title":"DMD genotypes and loss of ambulation in the CINRG Duchenne natural history study","volume":"26","author":"L Bello","year":"2016","journal-title":"Neuromuscular Disorders"},{"issue":"Suppl 1","key":"pone.0265879.ref068","doi-asserted-by":"crossref","first-page":"S108","DOI":"10.1016\/j.nmd.2019.06.257","article-title":"P.202 A composite prognostic score for time to loss of walking ability in Duchenne muscular dystrophy (DMD)","volume":"29","author":"N Goemans","year":"2019","journal-title":"Neuromuscular Disorders"},{"issue":"Suppl 3","key":"pone.0265879.ref069","doi-asserted-by":"crossref","first-page":"S864","DOI":"10.1016\/j.jval.2019.09.2456","article-title":"PRO126 A composite prognostic score for time to loss of walking ability in Duchenne muscular dystrophy (DMD)","volume":"22","author":"N Goemans","year":"2019","journal-title":"Value in Health"},{"issue":"10","key":"pone.0265879.ref070","doi-asserted-by":"crossref","first-page":"1275","DOI":"10.1177\/0883073814558120","article-title":"Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation","volume":"30","author":"S Kim","year":"2015","journal-title":"Journal of Child Neurology"},{"issue":"4","key":"pone.0265879.ref071","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1097\/00131402-200106000-00002","article-title":"Long-Term Steroid Therapy in Duchenne Muscular Dystrophy-Positive Results versus Side Effects","volume":"2","author":"U Schara","year":"2001","journal-title":"J Clin Neuromuscul Dis"},{"issue":"1","key":"pone.0265879.ref072","doi-asserted-by":"crossref","first-page":"99","DOI":"10.3233\/JND-140005","article-title":"Forty-Five Years of Duchenne Muscular Dystrophy in The Netherlands","volume":"1","author":"JC van den Bergen","year":"2014","journal-title":"J Neuromuscul Dis"},{"key":"pone.0265879.ref073","doi-asserted-by":"crossref","first-page":"91","DOI":"10.3233\/JND-140002","article-title":"Prolonged Ambulation in Duchenne Patients with a Mutation Amenable to Exon 44 Skipping","volume":"1","author":"JC van den Bergen","year":"2014","journal-title":"J Neuromuscul Dis"},{"key":"pone.0265879.ref074","first-page":"6","article-title":"Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits","author":"RT Wang","year":"2014","journal-title":"PLoS Curr"},{"issue":"Suppl 1","key":"pone.0265879.ref075","first-page":"14","article-title":"Benefits and adverse effects of glucocorticoids in males with Duchenne muscular dystrophy: A UK perspective. Developmental Medicine &","volume":"54","author":"V Ricotti","year":"2012","journal-title":"Child Neurology"},{"issue":"Suppl 1","key":"pone.0265879.ref076","doi-asserted-by":"crossref","first-page":"S21","DOI":"10.1016\/S1090-3798(11)70076-9","article-title":"2FC2.6 Benefits and adverse effects of glucocorticoids in boys with Duchenne Muscular Dystrophy: A UK perspective","volume":"15","author":"V Ricotti","year":"2011","journal-title":"European Journal of Paediatric Neurology"},{"issue":"9","key":"pone.0265879.ref077","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1016\/j.nmd.2011.06.970","article-title":"P4.5 Benefits and adverse effects of glucocorticoids in boys with Duchenne Muscular Dystrophy","volume":"21","author":"V Ricotti","year":"2011","journal-title":"Neuromuscular Disorders"},{"issue":"8","key":"pone.0265879.ref078","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1001\/archneur.1987.00520200022012","article-title":"Prednisone treatment in Duchenne muscular dystrophy. Long-term benefit","volume":"44","author":"S DeSilva","year":"1987","journal-title":"Arch Neurol"},{"issue":"8","key":"pone.0265879.ref079","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1111\/j.1468-1331.2004.00866.x","article-title":"Prednisolone therapy in Duchenne muscular dystrophy prolongs ambulation and prevents scoliosis","volume":"11","author":"O Yilmaz","year":"2004","journal-title":"Eur J Neurol"},{"issue":"8\u20139","key":"pone.0265879.ref080","first-page":"581","article-title":"Prednisolone therapy in Duchenne muscular dystrophy prolongs ambulation and prevents scoliosis","volume":"14","author":"O Yilmaz","year":"2004","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref081","first-page":"630","volume":"11","author":"O Tunca","year":"2001","journal-title":"Alternate day prednisone therapy in Duchenne muscular dystrophy"},{"issue":"1","key":"pone.0265879.ref082","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1067\/mpd.2001.109601","article-title":"Deflazacort treatment of Duchenne muscular dystrophy","volume":"138","author":"WD Biggar","year":"2001","journal-title":"J Pediatr"},{"issue":"Suppl 7","key":"pone.0265879.ref083","first-page":"IN1-2.002","article-title":"Age at First Symptoms\/Signs and Loss of Ambulation in Duchenne-Becker Muscular Dystrophy: Data from the MD STARNet (IN1-2.002)","volume":"80","author":"E Ciafaloni","year":"2013","journal-title":"Neurology"},{"issue":"1","key":"pone.0265879.ref084","doi-asserted-by":"crossref","first-page":"5","DOI":"10.3233\/PRM-160361","article-title":"Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STARnet","volume":"9","author":"E Ciafaloni","year":"2016","journal-title":"J Pediatr Rehabil Med"},{"issue":"5","key":"pone.0265879.ref085","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1016\/j.ajhg.2016.08.023","article-title":"Association study of exon variants in the NF-\u03baB and TGF\u03b2 pathways identifies CD40 as a modifier of Duchenne muscular dystrophy","volume":"99","author":"L Bello","year":"2016","journal-title":"The American Journal of Human Genetics"},{"issue":"2","key":"pone.0265879.ref086","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1002\/mus.27113","article-title":"Association of genetic mutations and loss of ambulation in childhood\u2010onset dystrophinopathy","volume":"63","author":"G Haber","year":"2021","journal-title":"Muscle & Nerve"},{"issue":"5","key":"pone.0265879.ref087","doi-asserted-by":"crossref","first-page":"341","DOI":"10.2217\/cer-2019-0171","article-title":"Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study","volume":"9","author":"E Mercuri","year":"2020","journal-title":"Journal of comparative effectiveness research"},{"issue":"9","key":"pone.0265879.ref088","doi-asserted-by":"crossref","first-page":"1193","DOI":"10.1002\/humu.23561","article-title":"DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype","volume":"39","author":"RT Wang","year":"2018","journal-title":"Hum Mutat"},{"issue":"3","key":"pone.0265879.ref089","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1016\/S0960-8966(98)00113-8","article-title":"Lower limb surgery in Duchenne muscular dystrophy","volume":"9","author":"J Forst","year":"1999","journal-title":"Neuromuscul Disord"},{"issue":"2","key":"pone.0265879.ref090","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1007\/BF00422837","article-title":"Importance of lower limb surgery in Duchenne muscular dystrophy","volume":"114","author":"R Forst","year":"1995","journal-title":"Arch Orthop Trauma Surg"},{"issue":"3","key":"pone.0265879.ref091","doi-asserted-by":"crossref","first-page":"269","DOI":"10.3233\/JND-150100","article-title":"Non-ambulant Duchenne patients theoretically treatable by exon 53 skipping have severe phenotype","volume":"2","author":"L Servais","year":"2015","journal-title":"J Neuromuscul Dis"},{"issue":"5","key":"pone.0265879.ref092","doi-asserted-by":"crossref","first-page":"444","DOI":"10.1212\/WNL.0b013e318227b164","article-title":"Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy","volume":"77","author":"DM Escolar","year":"2011","journal-title":"Neurology"},{"key":"pone.0265879.ref093","first-page":"S129","article-title":"DMD\u2013THERAPY: P. 284 ATL1102 treatment improves PUL2. 0 in non-ambulant boys with Duchenne muscular dystrophy compared to a natural history control","volume":"30","author":"G Tachas","year":"2020","journal-title":"Neuromuscular Disorders"},{"issue":"2","key":"pone.0265879.ref094","doi-asserted-by":"crossref","first-page":"e320","DOI":"10.1542\/peds.2006-1400","article-title":"Effect of long-term steroids on cough efficiency and respiratory muscle strength in patients with Duchenne muscular dystrophy","volume":"119","author":"AS Daftary","year":"2007","journal-title":"Pediatrics"},{"issue":"9","key":"pone.0265879.ref095","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1016\/j.nmd.2013.06.572","article-title":"P.11.11 Pulmonary function characteristics of boys with Duchenne Muscular Dystrophy by age groups, ambulatory status and steroid use","volume":"23","author":"RT Abresch","year":"2013","journal-title":"Neuromuscular Disorders"},{"issue":"1","key":"pone.0265879.ref096","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1002\/mus.23808","article-title":"The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures","volume":"48","author":"EK Henricson","year":"2013","journal-title":"Muscle Nerve"},{"issue":"11","key":"pone.0265879.ref097","doi-asserted-by":"crossref","first-page":"897","DOI":"10.1016\/j.nmd.2018.07.004","article-title":"Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids","volume":"28","author":"CM McDonald","year":"2018","journal-title":"Neuromuscul Disord"},{"issue":"Suppl 4","key":"pone.0265879.ref098","first-page":"30","article-title":"Steroid use delays but does not prevent loss of pulmonary function in patients with Duchene muscular dystrophy (DMD)","volume":"59","author":"E Henricson","year":"2017","journal-title":"Developmental Medicine & Child Neurology"},{"key":"pone.0265879.ref099","first-page":"A6883","article-title":"Steroid Use Delays but Does Not Prevent Loss of Pulmonary Function in Patients with Duchene Muscular Dystrophy (DMD)","volume":"195","author":"C McDonald","year":"2017","journal-title":"C105 Disorders of respiratory physiology and sleep in children"},{"issue":"10","key":"pone.0265879.ref100","doi-asserted-by":"crossref","first-page":"1309","DOI":"10.1007\/s00415-006-0212-1","article-title":"Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation","volume":"253","author":"S Pradhan","year":"2006","journal-title":"J Neurol"},{"issue":"6","key":"pone.0265879.ref101","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1001\/archneur.1991.00530180027012","article-title":"A Comparison of Daily and Alternate-Day Prednisone Therapy in the Treatment of Duchenne Muscular Dystrophy","volume":"48","author":"GM Fenichel","year":"1991","journal-title":"Archives of Neurology"},{"issue":"Suppl 16","key":"pone.0265879.ref102","doi-asserted-by":"crossref","first-page":"I4.009","DOI":"10.1212\/WNL.86.16_supplement.I4.009","article-title":"Effect of Deflazacort and Prednisone Versus Placebo on Pulmonary Function in Boys with Duchenne Muscular Dystrophy Who Have Lost Ambulation (I4.009)","volume":"86","author":"J Dubow","year":"2016","journal-title":"Neurology"},{"issue":"2","key":"pone.0265879.ref103","first-page":"69","article-title":"Respiratory function in ataluren-treated, nonambulatory patients with nonsense mutation Duchenne (nmDMD) muscular dystrophy from a long-term extension trial versus untreated patients from a natural history study","volume":"36","author":"GP Comi","year":"2017","journal-title":"Acta myologica: myopathies and cardiomyopathies"},{"issue":"2","key":"pone.0265879.ref104","first-page":"77","article-title":"Lung function in ataluren-treated, non-ambulatory patients with nonsense mutation Duchenne muscular dystrophy from a long-term extension trial versus untreated patients from a natural history study","volume":"16","author":"CM McDonald","year":"2016","journal-title":"Sinapse"},{"issue":"1","key":"pone.0265879.ref105","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1186\/s12931-019-1200-1","article-title":"Influence of \u03b22 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy","volume":"20","author":"EF Kelley","year":"2019","journal-title":"Respiratory Research"},{"issue":"1","key":"pone.0265879.ref106","doi-asserted-by":"crossref","first-page":"e000517","DOI":"10.1136\/bmjresp-2019-000517","article-title":"Early NIV is associated with accelerated lung function decline in Duchenne muscular dystrophy treated with glucocorticosteroids","volume":"7","author":"ME Angliss","year":"2020","journal-title":"BMJ open respiratory research"},{"issue":"5","key":"pone.0265879.ref107","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1002\/acn3.51046","article-title":"Genetic modifiers of respiratory function in Duchenne muscular dystrophy","volume":"7","author":"L Bello","year":"2020","journal-title":"Annals of clinical and translational neurology"},{"key":"pone.0265879.ref108","first-page":"S131","article-title":"DMD\u2013THERAPY: P. 291 Real-world evidence of eteplirsen treatment effects on Duchenne muscular dystrophy related health outcomes using claims data in the United States","volume":"30","author":"J Iff","year":"2020","journal-title":"Neuromuscular Disorders"},{"issue":"S1","key":"pone.0265879.ref109","first-page":"S132","article-title":"Ataluren delays loss of ambulation and decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy","volume":"30","author":"C McDonald","year":"2020","journal-title":"Neuromuscular Disorders Presented at the 25TH International Congress of the World Muscle Society (WMS), 2020 (Virtual Congress)"},{"issue":"SUPPL 1","key":"pone.0265879.ref110","first-page":"S53","article-title":"Ataluren delays loss of ambulation and decline in pulmonary function in patients with nonsense mutation duchenne muscular dystrophy","volume":"62","author":"F. McDonald CM","year":"2020","journal-title":"Muscle and Nerve"},{"issue":"6","key":"pone.0265879.ref111","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1016\/j.nmd.2011.02.016","article-title":"Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial","volume":"21","author":"GM Buyse","year":"2011","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref112","first-page":"A1765-A","article-title":"Comparison of Longitudinal Changes in Expiratory Respiratory Function Endpoints and Inspiratory Flow Reserve (IFR) in Patients with Duchenne Muscular Dystrophy (DMD)","author":"J Karafilidis","year":"2018","journal-title":"A47 NEUROMUSCULAR DISEASE AND RESPIRATION. American Thoracic Society International Conference Abstracts: American Thoracic Society"},{"issue":"2","key":"pone.0265879.ref113","doi-asserted-by":"crossref","first-page":"213","DOI":"10.3233\/JND-180351","article-title":"Eteplirsen treatment attenuates respiratory decline in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy","volume":"6","author":"N Khan","year":"2019","journal-title":"J Neuromuscul Dis"},{"issue":"15(S1)","key":"pone.0265879.ref114","article-title":"Eteplirsen-treatment attenuates respiratory decline in ambulatory and non-ambulatory patients with duchenne muscular dystrophy: Comparison with natural history cohorts","volume":"92","author":"N Khan","year":"2019","journal-title":"Neurology Conference: 71st Annual Meeting of the American Academy of Neurology, AAN"},{"issue":"S1","key":"pone.0265879.ref115","first-page":"S28","article-title":"Respiratory Function Decline in Eteplirsen-treated Patients Diverges From Natural History Comparators Over Time","volume":"6","author":"N Khan","journal-title":"J Neuromuscul Dis"},{"issue":"9","key":"pone.0265879.ref116","first-page":"828","article-title":"GP 112: Pulmonary function is stable through week 120 in patients with Duchenne muscular dystrophy (DMD) treated with exon-skipping drug eteplirsen in phase 2b study","volume":"24","author":"J Mendell","year":"2014","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref117","first-page":"S136","article-title":"Pulmonary function and safety results at week 120 of exon-skipping drug eteplirsen from the phase 2b study in patients with duchenne muscular dystrophy (DMD)","volume":"1","author":"J Mendell","year":"2014","journal-title":"J Neuromuscul Dis"},{"key":"pone.0265879.ref118","first-page":"S237","article-title":"Pulmonary function is stable in patients with duchenne muscular dystrophy (DMD) treated with exon-skipping drug eteplirsen in phase 2B study","volume":"76","author":"J Mendell","year":"2014","journal-title":"Annals of neurology"},{"key":"pone.0265879.ref119","first-page":"S63","article-title":"Overview of Eteplirsen Clinical Outcomes in Duchenne Muscular Dystrophy (DMD)","volume":"76","author":"J Mendell","year":"2014","journal-title":"Annals of Neurology"},{"key":"pone.0265879.ref120","first-page":"S237","article-title":"Eteplirsen in Duchenne Muscular Dystrophy (DMD): 144 week update on six-minute walk test (6MWT) and safety","volume":"76","author":"JR Mendell","year":"2014","journal-title":"Annals of Neurology"},{"key":"pone.0265879.ref121","doi-asserted-by":"crossref","first-page":"S16","DOI":"10.1016\/S1525-0016(16)33641-3","article-title":"C-2. Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety","volume":"23","author":"JR Mendell","year":"2015","journal-title":"Molecular Therapy"},{"issue":"10(S1)","key":"pone.0265879.ref122","article-title":"Results at 96 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne Muscular Dystrophy (DMD)(S6. 002)","volume":"82","author":"E Kaye","year":"2014","journal-title":"Neurology"},{"key":"pone.0265879.ref123","doi-asserted-by":"crossref","first-page":"S263","DOI":"10.1016\/j.nmd.2015.06.282","article-title":"Eteplirsen, a Phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): Clinical update","volume":"25","author":"E Kaye","year":"2015","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref124","first-page":"s105","article-title":"Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of duchenne muscular dystrophy (DMD)","volume":"19","author":"E Kaye","year":"2015","journal-title":"Annals of Neurology"},{"key":"pone.0265879.ref125","doi-asserted-by":"crossref","first-page":"S69","DOI":"10.1016\/S1090-3798(15)30228-2","article-title":"Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): 3.2 year update on six-minute walk test (6MWT), pulmonary function testing (PFT), and safety","volume":"19","author":"E Kaye","year":"2015","journal-title":"European journal of paediatric neurology"},{"issue":"4","key":"pone.0265879.ref126","doi-asserted-by":"crossref","first-page":"469","DOI":"10.3233\/JND-200548","article-title":"Comparison of long-term ambulatory function in patients with Duchenne muscular dystrophy treated with eteplirsen and matched natural history controls","volume":"8","author":"JR Mendell","year":"2021","journal-title":"J Neuromuscul Dis"},{"issue":"S20","key":"pone.0265879.ref127","first-page":"S415","article-title":"Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for duchenne muscular dystrophy (DMD): Longitudinal comparison to external controls on six-minute walk test (6MWT) and loss of ambulation (LOA)","volume":"80","author":"J Mendell","year":"2016","journal-title":"Annals of Neurology"},{"issue":"16S1","key":"pone.0265879.ref128","article-title":"Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for Duchenne Muscular Dystrophy (DMD): Longitudinal Comparison to External Controls on Six-Minute Walk Test (6MWT) and Loss of Ambulation (LOA)(S42. 004)","volume":"88","author":"J Mendell","year":"2017","journal-title":"Neurology"},{"issue":"16S1","key":"pone.0265879.ref129","article-title":"Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for Duchenne Muscular Dystrophy (DMD): Clinical Update and Longitudinal Comparison to External Controls on Six-Minute Walk Test (6MWT)(S28. 001)","volume":"86","author":"J Mendell","year":"2016","journal-title":"Neurology"},{"issue":"2","key":"pone.0265879.ref130","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1002\/ana.24555","article-title":"Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy","volume":"79","author":"JR Mendell","year":"2016","journal-title":"Annals of neurology"},{"issue":"1","key":"pone.0265879.ref131","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1002\/mus.26736","article-title":"Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials","volume":"61","author":"CM McDonald","year":"2020","journal-title":"Muscle Nerve"},{"issue":"S1","key":"pone.0265879.ref132","doi-asserted-by":"crossref","first-page":"S16","DOI":"10.1016\/S0960-8966(18)30338-9","article-title":"Effect of idebenone on bronchopulmonary adverse events and hospitalizations in patients with Duchene muscular dystrophy (DMD)","volume":"28","author":"C Lawrence","year":"2018","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref133","first-page":"S299","article-title":"Comparison of home-based versus hospital-based spirometry measurements in duchenne muscular dystrophy","volume":"5","author":"C Rummey","year":"2018","journal-title":"J Neuromuscul Dis"},{"issue":"1","key":"pone.0265879.ref134","doi-asserted-by":"crossref","first-page":"1","DOI":"10.2169\/internalmedicine1962.30.1","article-title":"Correlation between clinical features and deletions of the gene for dystrophin in Duchenne muscular dystrophy","volume":"30","author":"H Kanazawa","year":"1991","journal-title":"Jpn J Med"},{"issue":"6","key":"pone.0265879.ref135","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1111\/j.1365-2613.2006.00470.x","article-title":"The effects of glucocorticoid therapy on the inflammatory and dendritic cells in muscular dystrophies","volume":"87","author":"MR Hussein","year":"2006","journal-title":"Int J Exp Pathol"},{"issue":"4","key":"pone.0265879.ref136","doi-asserted-by":"crossref","first-page":"386","DOI":"10.1002\/mus.880170405","article-title":"Deflazacort in Duchenne dystrophy: study of long-term effect","volume":"17","author":"C Angelini","year":"1994","journal-title":"Muscle Nerve"},{"issue":"12","key":"pone.0265879.ref137","doi-asserted-by":"crossref","first-page":"1874","DOI":"10.1212\/WNL.41.12.1874","article-title":"Long\u2010term benefit from prednisone therapy in Duchenne muscular dystrophy","volume":"41","author":"GM Fenichel","year":"1991","journal-title":"Neurology"},{"issue":"6","key":"pone.0265879.ref138","doi-asserted-by":"crossref","first-page":"1001","DOI":"10.1002\/mus.24665","article-title":"Daily prednisone treatment in Duchenne muscular dystrophy in southwest China","volume":"52","author":"J Hu","year":"2015","journal-title":"Muscle Nerve"},{"issue":"1 Pt 1","key":"pone.0265879.ref139","first-page":"E67","article-title":"Effect of prednisone on protein metabolism in Duchenne dystrophy","volume":"268","author":"Z Rifai","year":"1995","journal-title":"Am J Physiol"},{"issue":"3","key":"pone.0265879.ref140","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/0960-8966(94)00048-E","article-title":"Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy","volume":"5","author":"E B\u00e4ckman","year":"1995","journal-title":"Neuromuscul Disord"},{"issue":"10","key":"pone.0265879.ref141","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1016\/S0960-8966(02)00180-3","article-title":"High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy","volume":"12","author":"AM Connolly","year":"2002","journal-title":"Neuromuscul Disord"},{"issue":"3 Pt 1","key":"pone.0265879.ref142","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1212\/WNL.43.3_Part_1.520","article-title":"Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months)","volume":"43","author":"RC Griggs","year":"1993","journal-title":"Neurology"},{"issue":"4","key":"pone.0265879.ref143","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/0960-8966(91)90099-E","article-title":"Steroids in Duchenne muscular dystrophy\u2014deflazacort trial","volume":"1","author":"LE Mesa","year":"1991","journal-title":"Neuromuscul Disord"},{"issue":"1","key":"pone.0265879.ref144","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1001\/archneur.62.1.128","article-title":"Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial","volume":"62","author":"EA Beenakker","year":"2005","journal-title":"Arch Neurol"},{"issue":"4","key":"pone.0265879.ref145","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1001\/archneur.1991.00530160047012","article-title":"Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group","volume":"48","author":"RC Griggs","year":"1991","journal-title":"Arch Neurol"},{"issue":"2","key":"pone.0265879.ref146","doi-asserted-by":"crossref","first-page":"222","DOI":"10.1002\/mus.10319","article-title":"Early prednisone treatment in Duchenne muscular dystrophy","volume":"27","author":"L Merlini","year":"2003","journal-title":"Muscle Nerve"},{"issue":"3","key":"pone.0265879.ref147","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1212\/WNL.0b013e318207afeb","article-title":"SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy","volume":"76","author":"E Pegoraro","year":"2011","journal-title":"Neurology"},{"issue":"8","key":"pone.0265879.ref148","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1212\/WNL.56.8.1075","article-title":"A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy","volume":"56","author":"GM Fenichel","year":"2001","journal-title":"Neurology"},{"issue":"5","key":"pone.0265879.ref149","doi-asserted-by":"crossref","first-page":"1225","DOI":"10.1212\/WNL.48.5.1225","article-title":"A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy","volume":"48","author":"G Fenichel","year":"1997","journal-title":"Neurology"},{"issue":"14","key":"pone.0265879.ref150","doi-asserted-by":"crossref","first-page":"973","DOI":"10.2217\/cer-2020-0095","article-title":"Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy","volume":"9","author":"C Campbell","year":"2020","journal-title":"Journal of comparative effectiveness research"},{"issue":"S1","key":"pone.0265879.ref151","first-page":"P110","article-title":"The role of dystrophin brain isoforms on early motor development and motor outcomes in young children with Duchenne muscular dystrophy","volume":"62","author":"M Chesshyre","year":"2020","journal-title":"Developmental Medicine and Child Neurology"},{"issue":"8","key":"pone.0265879.ref152","doi-asserted-by":"crossref","first-page":"982","DOI":"10.1001\/jamaneurol.2020.1264","article-title":"Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping: a phase 2 randomized clinical trial","volume":"77","author":"PR Clemens","year":"2020","journal-title":"JAMA neurology"},{"issue":"5","key":"pone.0265879.ref153","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1016\/j.nmd.2021.02.001","article-title":"Disease-modifying effects of edasalonexent, an NF-\u03baB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial","volume":"31","author":"RS Finkel","year":"2021","journal-title":"Neuromuscular Disorders"},{"issue":"15(S1)","key":"pone.0265879.ref154","article-title":"MoveDMD\u00ae: Positive Effects of Edasalonexent, an NF-\u03baB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension (S29. 006)","volume":"90","author":"R Finkel","year":"2018","journal-title":"Neurology"},{"issue":"15(S1)","key":"pone.0265879.ref155","article-title":"Edasalonexent, an NF-kB Inhibitor, Slows Longer-Term Disease Progression on Multiple Functional and MRI Assessments Compared to Control Period in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy (S51. 006)","volume":"92","author":"R Finkel","year":"2019","journal-title":"Neurology"},{"key":"pone.0265879.ref156","doi-asserted-by":"crossref","first-page":"S208","DOI":"10.1016\/j.nmd.2019.06.596","article-title":"O. 42Treatment of young boys with Duchenne muscular dystrophy with the NF-\u03baB inhibitor edasalonexent showed a slowing of disease progression as assessed by MRI and functional measures","volume":"29","author":"R Finkel","year":"2019","journal-title":"Neuromuscular Disorders"},{"issue":"S2","key":"pone.0265879.ref157","first-page":"S53","article-title":"MoveDMD, a Phase 2 with Open-Label Extension Study of Treatment of Young Boys with Duchenne Muscular Dystrophy with the NF-kappaB Inhibitor Edasalonexent Showed a Slowing of Disease Progression as Assessed by MRI and Functional Measures","volume":"6","author":"H Sweeney","year":"2019","journal-title":"J Neuromuscul Dis"},{"issue":"5","key":"pone.0265879.ref158","doi-asserted-by":"crossref","first-page":"683","DOI":"10.1590\/S0004-282X2010000500002","article-title":"Comparison of motor strength and function in patients with Duchenne muscular dystrophy with or without steroid therapy","volume":"68","author":"SL Parreira","year":"2010","journal-title":"Arq Neuropsiquiatr"},{"issue":"9","key":"pone.0265879.ref159","doi-asserted-by":"crossref","first-page":"810","DOI":"10.1016\/j.nmd.2013.06.599","article-title":"P.13.5 Magnetic resonance imaging and spectroscopy detect changes with age, corticosteroid treatment, and functional progression in DMD","volume":"23","author":"RJ Willcocks","year":"2013","journal-title":"Neuromuscular Disorders"},{"key":"pone.0265879.ref160","first-page":"S63","article-title":"DMD & BMD\u2013CLINICAL: P. 55 Prognostic factors for loss of ability to rise from supine in Duchenne muscular dystrophy (DMD)","volume":"30","author":"N Goemans","year":"2020","journal-title":"Neuromuscular Disorders"},{"issue":"6","key":"pone.0265879.ref161","doi-asserted-by":"crossref","first-page":"e0232870","DOI":"10.1371\/journal.pone.0232870","article-title":"Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration","volume":"15","author":"N Goemans","year":"2020","journal-title":"PloS one"},{"key":"pone.0265879.ref162","volume-title":"Clinical and Experimental Pharmacology and Physiology","author":"S Wilton","year":"2013"},{"issue":"9","key":"pone.0265879.ref163","doi-asserted-by":"crossref","first-page":"A718","DOI":"10.1016\/j.jval.2017.08.1918","article-title":"Deflazacort or Prednisone Treatment for Duchenne Muscular Dystrophy: A Meta-Analysis of Disease Progression Rates in Recent Multicenter Clinical Trials","volume":"20","author":"JE Signorovitch","year":"2017","journal-title":"Value in Health"},{"issue":"Suppl 15","key":"pone.0265879.ref164","first-page":"P1","article-title":"Deflazacort or Prednisone Treatment for Duchenne Muscular Dystrophy: A Meta- Analysis of Disease Progression Rates in Recent Multicenter Clinical Trials (P1.6\u2013066)","volume":"92","author":"J Signorovitch","year":"2019","journal-title":"Neurology"},{"issue":"SUPPL 2","key":"pone.0265879.ref165","first-page":"S13","article-title":"Deflazacort or prednisone treatment for duchenne muscular dystrophy: A meta-analysis of disease progression rates in two multicenter clinical trials","volume":"60","author":"J Signorovitch","year":"2019","journal-title":"Muscle and Nerve"},{"issue":"Supplement 23","key":"pone.0265879.ref166","first-page":"S128","article-title":"Deflazacort or prednisone treatment for duchenne muscular dystrophy: A meta-analysis of disease progression rates in two multicenter clinical trials","volume":"86","author":"J Signorovitch","year":"2019","journal-title":"Annals of Neurology"},{"issue":"Supplement 1","key":"pone.0265879.ref167","first-page":"S65","article-title":"DMD & BMD\u2014CLINICAL: P.59 Functional progression in young DMD","volume":"30","author":"VA Gupta","year":"2020","journal-title":"Neuromuscular Disorders"},{"issue":"S1","key":"pone.0265879.ref168","first-page":"S137","article-title":"Longitudinal Effect of Drisapersen Versus Historical Controls on Ambulation in Duchenne Muscular Dystrophy","volume":"3","author":"N Goemans","year":"2016","journal-title":"J Neuromuscul Dis"},{"issue":"9","key":"pone.0265879.ref169","doi-asserted-by":"crossref","first-page":"e0161955","DOI":"10.1371\/journal.pone.0161955","article-title":"Long-term efficacy, safety, and pharmacokinetics of drisapersen in Duchenne muscular dystrophy: results from an open-label extension study","volume":"11","author":"NM Goemans","year":"2016","journal-title":"PloS one"},{"issue":"6","key":"pone.0265879.ref170","doi-asserted-by":"crossref","first-page":"698","DOI":"10.1136\/jnnp-2012-303902","article-title":"Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy","volume":"84","author":"V Ricotti","year":"2013","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"2","key":"pone.0265879.ref171","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1111\/dmcn.13590","article-title":"Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials.","volume":"60","author":"A Schreiber","year":"2018","journal-title":"Developmental Medicine & Child Neurology"},{"issue":"Suppl 1","key":"pone.0265879.ref172","doi-asserted-by":"crossref","first-page":"e139","DOI":"10.1016\/j.rehab.2015.07.331","article-title":"Promoting the use of Motor Function Measure (MFM) as outcome measure in patients with Duchenne Muscular Dystrophy (DMD) treated by corticosteroids","volume":"58","author":"A Schreiber-Bontemps","year":"2015","journal-title":"Annals of Physical and Rehabilitation Medicine"},{"issue":"S6","key":"pone.0265879.ref173","first-page":"22","article-title":"The natural history of Duchenne muscular dystrophy with corticosteroids using the Motor Function Measure","volume":"58","author":"A Schreiber","year":"2016","journal-title":"Developmental Medicine & Child Neurology"},{"issue":"Suppl 1","key":"pone.0265879.ref174","doi-asserted-by":"crossref","first-page":"S105","DOI":"10.1016\/j.nmd.2019.06.247","article-title":"P.192 The 100 meter timed test: responsiveness to change, predicting loss of ambulation, and data-driven phenotypes","volume":"29","author":"L Alfano","year":"2019","journal-title":"Neuromuscular Disorders"},{"issue":"10","key":"pone.0265879.ref175","doi-asserted-by":"crossref","first-page":"e0164684","DOI":"10.1371\/journal.pone.0164684","article-title":"Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy","volume":"11","author":"N Goemans","year":"2016","journal-title":"PLOS ONE"},{"issue":"Suppl 1","key":"pone.0265879.ref176","first-page":"S196","article-title":"Development of a prognostic model for 1-year change in timed 4 stair-climb in duchenne patients","volume":"5","author":"N Goemans","year":"2018","journal-title":"J Neuromuscul Dis"},{"issue":"9","key":"pone.0265879.ref177","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1016\/j.nmd.2014.06.227","article-title":"T.P.4: Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36month changes","volume":"24","author":"ES Mazzone","year":"2014","journal-title":"Neuromuscular Disorders"},{"issue":"Suppl 1","key":"pone.0265879.ref178","first-page":"S1","article-title":"Meta-analyses of deflazacort vs prednisone\/prednisolone in patients with nonsense mutation duchenne muscular dystrophy","volume":"58","author":"O Shieh P","year":"2018","journal-title":"Muscle & Nerve"},{"issue":"Suppl 15","key":"pone.0265879.ref179","doi-asserted-by":"crossref","first-page":"P2.325","DOI":"10.1212\/WNL.90.15_supplement.P2.325","article-title":"Meta-Analysis of Deflazacort vs Prednisone\/Prednisolone in Patients with Duchenne Muscular Dystrophy (P2.325)","volume":"90","author":"B Darras","year":"2018","journal-title":"Neurology"},{"issue":"4","key":"pone.0265879.ref180","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1002\/mus.24332","article-title":"Ataluren treatment of patients with nonsense mutation dystrophinopathy","volume":"50","author":"K Bushby","year":"2014","journal-title":"Muscle & nerve"},{"issue":"Suppl 1","key":"pone.0265879.ref181","first-page":"S21","article-title":"Safety and Efficacy of low-dose ataluren in boys with nonsense mutation dystrophinopathy","volume":"38","author":"JK Mah","year":"2011","journal-title":"Canadian Journal of Neurological Sciences \/ Journal Canadien des Sciences Neurologiques"},{"issue":"9","key":"pone.0265879.ref182","doi-asserted-by":"crossref","first-page":"804","DOI":"10.1016\/j.nmd.2013.06.580","article-title":"innonsense mutation Duchenne muscular dystrophy\u2013results of a post hoc analysis of change in %-predicted 6-min walk distance","volume":"23","author":"CM McDonald","year":"2013","journal-title":"Neuromuscular Disorders"},{"issue":"9","key":"pone.0265879.ref183","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1016\/j.nmd.2014.06.228","article-title":"T.P.5: Timed function tests and other physical function outcomes in Ataluren-treated patients with nonsense mutation Duchenne Muscular Dystrophy (nmDMD)","volume":"24","author":"CM McDonald","year":"2014","journal-title":"Neuromuscular Disorders"},{"issue":"Suppl 18","key":"pone.0265879.ref184","first-page":"S236","volume":"76","author":"CM McDonald","year":"2014","journal-title":"Timed function tests and other physical function outcomes in ataluren-treated patients with nonsense mutation duchenne muscular dystrophy (nmDMD)"},{"issue":"10101","key":"pone.0265879.ref185","doi-asserted-by":"crossref","first-page":"1489","DOI":"10.1016\/S0140-6736(17)31611-2","article-title":"a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"390","author":"CM McDonald","year":"2017","journal-title":"Lancet"},{"issue":"Suppl 15","key":"pone.0265879.ref186","doi-asserted-by":"crossref","first-page":"S51.004","DOI":"10.1212\/WNL.92.15_supplement.S51.004","article-title":"Preservation of Function over time as Measured by North Star Ambulatory Assessment in Ambulatory Boys with Nonsense Mutation Muscular Dystrophy Treated with Ataluren (S51.004)","volume":"92","author":"C McDonald","year":"2019","journal-title":"Neurology"},{"issue":"SUPPL 1","key":"pone.0265879.ref187","first-page":"S58","article-title":"Preservation of function over time as measured by North Star ambulatory assessment in ambulatory boys with nonsense mutation muscular dystrophy treated with ataluren","volume":"60","author":"C McDonald","year":"2019","journal-title":"Muscle and Nerve"},{"issue":"16 Supplement","key":"pone.0265879.ref188","doi-asserted-by":"crossref","first-page":"I15.008","DOI":"10.1212\/WNL.86.16_supplement.I15.008","article-title":"Results of North Star Ambulatory Assessments (NSAA) in the Phase 3 Ataluren Confirmatory Trial in Patients with Nonsense Mutation Duchenne Muscular Dystrophy (ACT DMD) (I15.008)","volume":"86","author":"K Bushby","year":"2016","journal-title":"Neurology"},{"issue":"Suppl 1","key":"pone.0265879.ref189","doi-asserted-by":"crossref","first-page":"e237","DOI":"10.1016\/j.ejpn.2017.04.1267","article-title":"Use of \u2265 5-second threshold in baseline time to stand from supine to predict disease progression in Duchenne muscular dystrophy","volume":"21","author":"CM McDonald","year":"2017","journal-title":"European Journal of Paediatric Neurology"},{"issue":"Suppl 1","key":"pone.0265879.ref190","doi-asserted-by":"crossref","first-page":"S12","DOI":"10.1016\/S0960-8966(18)30324-9","article-title":"Use of a \u22655-second threshold in baseline time to stand from supine to predict disease progression in Duchenne muscular dystrophy","volume":"28","author":"CM McDonald","year":"2018","journal-title":"Neuromuscular Disorders"},{"issue":"Suppl 1","key":"pone.0265879.ref191","first-page":"S199","article-title":"Use of a >\/ = 5-second threshold in baseline time to stand from supine to predict progression in DMD","volume":"5","author":"CM McDonald","year":"2018","journal-title":"J Neuromuscul Dis"},{"issue":"Supplement 23","key":"pone.0265879.ref192","first-page":"S126","article-title":"Timed-function test data in patients with duchenne muscular dystrophy from the strategic targeting of registries and international database of excellence (STRIDE) registry and the CINRG natural history study: A matched cohort analysis","volume":"86","author":"E Mercuri","year":"2019","journal-title":"Annals of Neurology"},{"issue":"Supplement 23","key":"pone.0265879.ref193","first-page":"S127","article-title":"Age at loss of ambulation in patients with duchenne muscular dystrophy from the stride registry and the CINRG natural history study: A matched cohort analysis","volume":"86","author":"F Muntoni","year":"2019","journal-title":"Annals of Neurology"},{"issue":"6","key":"pone.0265879.ref194","doi-asserted-by":"crossref","first-page":"e0218683","DOI":"10.1371\/journal.pone.0218683","article-title":"Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53","volume":"14","author":"C Brogna","year":"2019","journal-title":"PLOS ONE"},{"issue":"Suppl 1","key":"pone.0265879.ref195","doi-asserted-by":"crossref","first-page":"S91","DOI":"10.1016\/j.nmd.2019.06.204","article-title":"P.148 Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53","volume":"29","author":"C Brogna","year":"2019","journal-title":"Neuromuscular Disorders"},{"issue":"2","key":"pone.0265879.ref196","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1002\/acn3.50978","article-title":"Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy","volume":"7","author":"H Komaki","year":"2020","journal-title":"Annals of Clinical and Translational Neurology"},{"issue":"13","key":"pone.0265879.ref197","doi-asserted-by":"crossref","first-page":"E1312","DOI":"10.1212\/WNL.0000000000008168","article-title":"Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function","volume":"93","author":"EP Hoffman","year":"2019","journal-title":"Neurology"},{"issue":"9","key":"pone.0265879.ref198","doi-asserted-by":"crossref","first-page":"e1003222","DOI":"10.1371\/journal.pmed.1003222","article-title":"Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study","volume":"17","author":"EC Smith","year":"2020","journal-title":"PLoS Med"},{"issue":"4","key":"pone.0265879.ref199","doi-asserted-by":"crossref","first-page":"293","DOI":"10.3233\/JND-170280","article-title":"Clinical outcomes in Duchenne muscular dystrophy: a study of 5345 patients from the TREAT-NMD DMD global database","volume":"4","author":"Z Koeks","year":"2017","journal-title":"J Neuromuscul Dis"},{"issue":"10","key":"pone.0265879.ref200","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1016\/S1474-4422(14)70195-4","article-title":"Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study","volume":"13","author":"T Voit","year":"2014","journal-title":"Lancet Neurol"},{"issue":"S14","key":"pone.0265879.ref201","article-title":"Drisapersen: An overview of the EXON51 skipping antisense oligonucleotide clinical program to date in duchenne muscular dystrophy (DMD)","volume":"84","author":"C McDonald","year":"2015","journal-title":"Neurology Conference: 67th American Academy of Neurology Annual Meeting, AAN"},{"issue":"9\u201310","key":"pone.0265879.ref202","first-page":"922","article-title":"Drisapersen: An overview of the clinical programme to date in Duchenne Muscular Dystrophy (DMD)","volume":"24","author":"C McDonald","year":"2014","journal-title":"Neuromuscular Disorders"},{"issue":"3","key":"pone.0265879.ref203","doi-asserted-by":"crossref","first-page":"189","DOI":"10.3233\/JND-170245","article-title":"Efficacy of Idebenone to Preserve Respiratory Function above Clinically Meaningful Thresholds for Forced Vital Capacity (FVC) in Patients with Duchenne Muscular Dystrophy","volume":"4","author":"OH Mayer","year":"2017","journal-title":"J Neuromuscul Dis"}],"updated-by":[{"DOI":"10.1371\/journal.pone.0315682","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2024,12,9]],"date-time":"2024-12-09T00:00:00Z","timestamp":1733702400000}}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0265879","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,9,20]],"date-time":"2024-09-20T21:00:41Z","timestamp":1726866041000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0265879"}},"subtitle":[],"editor":[{"given":"Otavio R.","family":"Coelho-Filho","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2022,3,25]]},"references-count":203,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2022,3,25]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0265879","relation":{"has-review":[{"id-type":"doi","id":"10.32388\/Q1WWRQ","asserted-by":"object"}]},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,3,25]]}}}