{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T04:06:54Z","timestamp":1791259614979,"version":"4.1.0"},"reference-count":180,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,7,22]],"date-time":"2022-07-22T00:00:00Z","timestamp":1658448000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Pharmacol."],"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of functional dystrophin protein, which results in chronic damage of muscle fibers during contraction, thus leading to deterioration of muscle quality and loss of muscle mass over time. Although there is currently no cure for DMD, improvements in treatment care and management could delay disease progression and improve quality of life, thereby prolonging life expectancy for these patients. Furthermore, active research efforts are ongoing to develop therapeutic strategies that target dystrophin deficiency, such as gene replacement therapies, exon skipping, and readthrough therapy, as well as strategies that target secondary pathology of DMD, such as novel anti-inflammatory compounds, myostatin inhibitors, and cardioprotective compounds. Furthermore, longitudinal modeling approaches have been used to characterize the progression of MRI and functional endpoints for predictive purposes to inform Go\/No Go decisions in drug development. This review showcases approved drugs or drug candidates along their development paths and also provides information on primary endpoints and enrollment size of Ph2\/3 and Ph3 trials in the DMD space.<\/jats:p>","DOI":"10.3389\/fphar.2022.950651","type":"journal-article","created":{"date-parts":[[2022,7,22]],"date-time":"2022-07-22T06:10:44Z","timestamp":1658470244000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":73,"title":["Drug development progress in duchenne muscular dystrophy"],"prefix":"10.3389","volume":"13","author":[{"given":"Jiexin","family":"Deng","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Junshi","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keli","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhigang","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2022,7,22]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"293","DOI":"10.1002\/humu.20918","article-title":"Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations","volume":"30","author":"Aartsma-Rus","year":"2009","journal-title":"Hum. Mutat."},{"key":"B2","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1136\/jmedgenet-2015-103387","article-title":"The importance of genetic diagnosis for Duchenne muscular dystrophy","volume":"53","author":"Aartsma-Rus","year":"2016","journal-title":"J. Med. Genet."},{"key":"B3","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1089\/nat.2016.0657","article-title":"FDA approves eteplirsen for duchenne muscular dystrophy: The next chapter in the eteplirsen saga","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid. Ther."},{"key":"B4","doi-asserted-by":"publisher","first-page":"251","DOI":"10.1089\/nat.2017.0682","article-title":"Development of exon skipping therapies for duchenne muscular dystrophy: A critical review and a perspective on the outstanding issues","volume":"27","author":"Aartsma-Rus","year":"2017","journal-title":"Nucleic Acid. Ther."},{"key":"B5","doi-asserted-by":"publisher","first-page":"19079","DOI":"10.1073\/pnas.1013077107","article-title":"Sildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophy","volume":"107","author":"Adamo","year":"2010","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B6","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1152\/physrev.00007.2015","article-title":"Absence of dystrophin disrupts skeletal muscle signaling: Roles of Ca2+, reactive oxygen species, and nitric oxide in the development of muscular dystrophy","volume":"96","author":"Allen","year":"2016","journal-title":"Physiol. Rev."},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1371\/currents.md.2cc69a1dae4be7dfe2bcb420024ea865","article-title":"A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy","volume":"5","author":"Allen","year":"2013","journal-title":"PLoS Curr."},{"key":"B8","doi-asserted-by":"publisher","first-page":"386","DOI":"10.1002\/mus.880170405","article-title":"Deflazacort in duchenne dystrophy: Study of long-term effect","volume":"17","author":"Angelini","year":"1994","journal-title":"Muscle Nerve"},{"key":"B9","first-page":"9","article-title":"Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy","volume":"31","author":"Angelini","year":"2012","journal-title":"Acta Myol."},{"key":"B10","doi-asserted-by":"publisher","first-page":"51","DOI":"10.2147\/odrr.s55677","article-title":"Drugs in development and dietary approach for Duchenne muscular dystrophy","volume":"5","author":"Angelini","year":"","journal-title":"Orphan Drugs Res. Rev."},{"key":"B11","article-title":"Management and clinical trials in Duchenne dystrophy","volume-title":"Duchenne muscular dystrophy: Symptoms, management and prognosis","author":"Angelini","year":""},{"key":"B12","doi-asserted-by":"publisher","first-page":"424","DOI":"10.1002\/mus.20812","article-title":"The role of corticosteroids in muscular dystrophy: A critical appraisal","volume":"36","author":"Angelini","year":"2007","journal-title":"Muscle Nerve"},{"key":"B13","doi-asserted-by":"publisher","first-page":"3547","DOI":"10.1093\/brain\/awr291","article-title":"Dystrophin quantification and clinical correlations in becker muscular dystrophy: Implications for clinical trials","volume":"134","author":"Anthony","year":"2011","journal-title":"Brain"},{"key":"B14","doi-asserted-by":"publisher","first-page":"620","DOI":"10.1097\/PHM.0b013e3181e72207","article-title":"Duchenne muscular dystrophy: The effect of glucocorticoids on ventilator use and ambulation","volume":"89","author":"Bach","year":"2010","journal-title":"Am. J. Phys. Med. Rehabil."},{"key":"B15","doi-asserted-by":"publisher","first-page":"375","DOI":"10.1172\/JCI7866","article-title":"Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice","volume":"104","author":"Barton-Davis","year":"1999","journal-title":"J. Clin. Invest."},{"key":"B16","article-title":"Acceleration of clinical trial design in duchenne muscular dystrophy building a model-based clinical trial simulation tool","volume":"2022","author":"Belfiore-Oshan","year":"2022","journal-title":"MDA Conf."},{"key":"B17","article-title":"Safety and efficacy of PF-06939926 gene therapy in boys with duchenne muscular dystrophy: Update on data from the phase 1b study","author":"Belluscio","year":"2021","journal-title":"Muscular Dystrophy Assoc."},{"key":"B18","doi-asserted-by":"publisher","first-page":"643","DOI":"10.1016\/j.nmd.2016.07.002","article-title":"Histological effects of givinostat in boys with Duchenne muscular dystrophy","volume":"26","author":"Bettica","year":"2016","journal-title":"Neuromuscul. Disord."},{"key":"B19","doi-asserted-by":"publisher","first-page":"484","DOI":"10.1002\/cpdd.386","article-title":"Safety, tolerability, pharmacokinetics, and pharmacodynamics of domagrozumab (PF-06252616), an antimyostatin monoclonal antibody, in healthy subjects","volume":"7","author":"Bhattacharya","year":"2018","journal-title":"Clin. Pharmacol. Drug Dev."},{"key":"B20","doi-asserted-by":"publisher","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":"Biggar","year":"2006","journal-title":"Neuromuscul. Disord."},{"key":"B21","doi-asserted-by":"publisher","first-page":"476","DOI":"10.1016\/j.nmd.2004.05.001","article-title":"Deflazacort in duchenne muscular dystrophy: A comparison of two different protocols","volume":"14","author":"Biggar","year":"2004","journal-title":"Neuromuscul. Disord."},{"key":"B22","doi-asserted-by":"publisher","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":"Birnkrant","year":"","journal-title":"Lancet. Neurol."},{"key":"B23","doi-asserted-by":"publisher","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and management of duchenne muscular dystrophy, part 1: Diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management","volume":"17","author":"Birnkrant","year":"","journal-title":"Lancet. Neurol."},{"key":"B24","doi-asserted-by":"publisher","first-page":"445","DOI":"10.1016\/S1474-4422(18)30026-7","article-title":"Diagnosis and management of duchenne muscular dystrophy, part 3: Primary care, emergency management, psychosocial care, and transitions of care across the lifespan","volume":"17","author":"Birnkrant","year":"","journal-title":"Lancet. Neurol."},{"key":"B25","doi-asserted-by":"publisher","first-page":"395","DOI":"10.1002\/humu.22758","article-title":"The TREAT-NMD DMD global database: Analysis of more than 7, 000 duchenne muscular dystrophy mutations","volume":"36","author":"Bladen","year":"2015","journal-title":"Hum. Mutat."},{"key":"B26","doi-asserted-by":"publisher","first-page":"1344","DOI":"10.1002\/1097-4598(200009)23:9<1344::aid-mus4>3.0.co;2-f","article-title":"A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy","volume":"23","author":"Bonifati","year":"2000","journal-title":"Muscle Nerve"},{"key":"B27","doi-asserted-by":"publisher","first-page":"634","DOI":"10.1016\/j.nmd.2019.06.598","article-title":"238th ENMC international workshop: Updating management recommendations of cardiac dystrophinopathyHoofddorp, The Netherlands, 30 november - 2 december 2018","volume":"29","author":"Bourke","year":"2019","journal-title":"Neuromuscul. Disord."},{"key":"B28","doi-asserted-by":"publisher","first-page":"S72","DOI":"10.1542\/peds.2018-0333I","article-title":"Cardiac management of the patient with duchenne muscular dystrophy","volume":"142","author":"Buddhe","year":"2018","journal-title":"Pediatrics"},{"key":"B29","doi-asserted-by":"publisher","first-page":"477","DOI":"10.1002\/mus.24332","article-title":"Ataluren treatment of patients with nonsense mutation dystrophinopathy","volume":"50","author":"Bushby","year":"2014","journal-title":"Muscle Nerve"},{"key":"B30","doi-asserted-by":"publisher","first-page":"912","DOI":"10.1002\/ppul.22688","article-title":"Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy","volume":"48","author":"Buyse","year":"2013","journal-title":"Pediatr. Pulmonol."},{"key":"B31","doi-asserted-by":"publisher","first-page":"1748","DOI":"10.1016\/S0140-6736(15)60025-3","article-title":"Efficacy of idebenone on respiratory function in patients with duchenne muscular dystrophy not using glucocorticoids (DELOS): A double-blind randomised placebo-controlled phase 3 trial","volume":"385","author":"Buyse","year":"2015","journal-title":"Lancet"},{"key":"B32","article-title":"Capricor therapeutics announces positive final data from its phase 2 HOPE-2 trial in patients with duchenne muscular dystrophy treated with CAP-1002","year":"2021","journal-title":"Capricor Ther."},{"key":"B33","doi-asserted-by":"publisher","first-page":"377","DOI":"10.1016\/j.ccm.2018.01.004","article-title":"Muscular dystrophies","volume":"39","author":"Carter","year":"2018","journal-title":"Clin. Chest Med."},{"key":"B34","doi-asserted-by":"publisher","first-page":"e2146","DOI":"10.1212\/WNL.0000000000005680","article-title":"Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production","volume":"90","author":"Charleston","year":"2018","journal-title":"Neurology"},{"key":"B35","doi-asserted-by":"publisher","first-page":"595","DOI":"10.1016\/S0140-6736(11)60756-3","article-title":"Exon skipping and dystrophin restoration in patients with duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study","volume":"378","author":"Cirak","year":"2011","journal-title":"Lancet"},{"key":"B36","doi-asserted-by":"publisher","first-page":"1","DOI":"10.3233\/JND-220811","article-title":"Long-term functional efficacy and safety of viltolarsen in patients with duchenne muscular dystrophy","author":"Clemens","year":"2022","journal-title":"J. Neuromuscul. Dis."},{"key":"B37","doi-asserted-by":"publisher","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":"Clemens","year":"2020","journal-title":"JAMA Neurol."},{"key":"B38","doi-asserted-by":"publisher","first-page":"554","DOI":"10.1007\/bf02388063","article-title":"Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle","volume":"25","author":"Clerk","year":"1993","journal-title":"Histochem. J."},{"key":"B39","doi-asserted-by":"publisher","first-page":"813","DOI":"10.1038\/ng1810","article-title":"A mutation creating a potential illegitimate microRNA target site in the myostatin gene affects muscularity in sheep","volume":"38","author":"Clop","year":"2006","journal-title":"Nat. Genet."},{"key":"B40","doi-asserted-by":"publisher","first-page":"140","DOI":"10.1016\/j.phrs.2018.09.007","article-title":"Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug","volume":"136","author":"Conklin","year":"2018","journal-title":"Pharmacol. Res."},{"key":"B41","doi-asserted-by":"publisher","first-page":"79","DOI":"10.2119\/molmed.2013.00011","article-title":"Preclinical studies in the mdx mouse model of duchenne muscular dystrophy with the histone deacetylase inhibitor givinostat","volume":"19","author":"Consalvi","year":"2013","journal-title":"Mol. Med."},{"key":"B42","doi-asserted-by":"publisher","first-page":"511","DOI":"10.1177\/0884533615586202","article-title":"Nutrition considerations in duchenne muscular dystrophy","volume":"30","author":"Davis","year":"2015","journal-title":"Nutr. Clin. Pract."},{"key":"B43","doi-asserted-by":"publisher","first-page":"717","DOI":"10.1016\/s0092-8674(00)80532-2","article-title":"Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy","volume":"90","author":"Deconinck","year":"1997","journal-title":"Cell"},{"key":"B44","volume-title":"Population longitudinal analysis of magnetic resonance imaging (MRI) and time to climb four stairs (4SC) in DMD patients receiving domagrozumab (conference presentation)","author":"Deng","year":"2021"},{"key":"B45","doi-asserted-by":"publisher","first-page":"105","DOI":"10.1186\/s13023-019-1066-9","article-title":"Effect and safety of treatment with ACE-inhibitor Enalapril and beta-blocker metoprolol on the onset of left ventricular dysfunction in Duchenne muscular dystrophy - A randomized, double-blind, placebo-controlled trial","volume":"14","author":"Dittrich","year":"2019","journal-title":"Orphanet J. Rare Dis."},{"key":"B46","article-title":"A phase 1b\/2 study of the anti-myostatin adnectin RG6206 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy: A 120-week treatment update","author":"Dreghici","year":"2019","journal-title":"2019 European Academy of Neurology (ean) conference"},{"key":"B47","doi-asserted-by":"publisher","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","article-title":"Systemic AAV micro-dystrophin gene therapy for duchenne muscular dystrophy","volume":"26","author":"Duan","year":"2018","journal-title":"Mol. Ther."},{"key":"B48","doi-asserted-by":"publisher","first-page":"240","DOI":"10.1177\/088307388900400402","article-title":"The duchenne dystrophy story: From phenotype to gene and potential treatment","volume":"4","author":"Dubowitz","year":"1989","journal-title":"J. Child. Neurol."},{"key":"B49","doi-asserted-by":"publisher","first-page":"624","DOI":"10.1002\/mus.10341","article-title":"Gentamicin fails to increase dystrophin expression in dystrophin-deficient muscle","volume":"27","author":"Dunant","year":"2003","journal-title":"Muscle Nerve"},{"key":"B50","doi-asserted-by":"publisher","first-page":"165","DOI":"10.1093\/oxfordjournals.bmb.a071633","article-title":"Intermediary metabolism of muscle in Duchenne muscular dystrophy","volume":"36","author":"Ellis","year":"1980","journal-title":"Br. Med. Bull."},{"key":"B51","article-title":"European public assessment report (EPAR) for translarna","author":"Ema","year":"2021","journal-title":"European Medicines Agency"},{"key":"B52","volume-title":"Exondys: EPAR - refusal public assessment report","author":"Ema","year":"2018"},{"key":"B53","doi-asserted-by":"publisher","first-page":"687","DOI":"10.1016\/S0140-6736(02)07815-7","article-title":"The muscular dystrophies","volume":"359","author":"Emery","year":"2002","journal-title":"Lancet"},{"key":"B54","article-title":"FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation","year":"2020","journal-title":"FDA News Release"},{"key":"B55","article-title":"FDA grants accelerated approval to first drug for Duchenne muscular dystrophy","year":"2016","journal-title":"FDA News Release"},{"key":"B56","article-title":"FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation","year":"2019","journal-title":"FDA News Release"},{"key":"B57","doi-asserted-by":"publisher","first-page":"456","DOI":"10.1164\/rccm.200307-885ST","article-title":"Respiratory care of the patient with duchenne muscular dystrophy: ATS consensus statement","volume":"170","author":"Finder","year":"2004","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"B58","doi-asserted-by":"publisher","first-page":"e81302","DOI":"10.1371\/journal.pone.0081302","article-title":"Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy","volume":"8","author":"Finkel","year":"2013","journal-title":"PLoS One"},{"key":"B59","doi-asserted-by":"publisher","first-page":"735","DOI":"10.1152\/physiolgenomics.00134.2017","article-title":"Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone","volume":"50","author":"Fiorillo","year":"2018","journal-title":"Physiol. Genomics"},{"key":"B60","doi-asserted-by":"publisher","first-page":"e2270","DOI":"10.1212\/WNL.0000000000009233","article-title":"Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy","volume":"94","author":"Frank","year":"2020","journal-title":"Neurology"},{"key":"B61","doi-asserted-by":"publisher","first-page":"309","DOI":"10.1016\/j.bbrc.2012.12.105","article-title":"Myostatin acts as an autocrine\/paracrine negative regulator in myoblast differentiation from human induced pluripotent stem cells","volume":"431","author":"Gao","year":"2013","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"B62","doi-asserted-by":"publisher","first-page":"1223","DOI":"10.1002\/cphy.c140048","article-title":"The dystrophin complex: Structure, function, and implications for therapy","volume":"5","author":"Gao","year":"2015","journal-title":"Compr. Physiol."},{"key":"B63","doi-asserted-by":"publisher","first-page":"4225","DOI":"10.1093\/hmg\/ddv155","article-title":"How much dystrophin is enough: The physiological consequences of different levels of dystrophin in the mdx mouse","volume":"24","author":"Godfrey","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"B64","doi-asserted-by":"publisher","first-page":"e1381","DOI":"10.1212\/WNL.0000000000010170","article-title":"Suitability of external controls for drug evaluation in Duchenne muscular dystrophy","volume":"95","author":"Goemans","year":"2020","journal-title":"Neurology"},{"key":"B65","doi-asserted-by":"publisher","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":"Goemans","year":"2016","journal-title":"PLoS One"},{"key":"B66","doi-asserted-by":"publisher","first-page":"729","DOI":"10.1016\/s0092-8674(00)80533-4","article-title":"Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: A model for duchenne muscular dystrophy","volume":"90","author":"Grady","year":"1997","journal-title":"Cell"},{"key":"B67","doi-asserted-by":"publisher","first-page":"1787","DOI":"10.1093\/hmg\/9.12.1787","article-title":"A biochemical basis for the inherited susceptibility to aminoglycoside ototoxicity","volume":"9","author":"Guan","year":"2000","journal-title":"Hum. Mol. Genet."},{"key":"B68","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1016\/j.nmd.2014.11.011","article-title":"European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene","volume":"25","author":"Haas","year":"2015","journal-title":"Neuromuscul. Disord."},{"key":"B69","doi-asserted-by":"publisher","first-page":"e003911","DOI":"10.1161\/JAHA.116.003911","article-title":"Tadalafil treatment delays the onset of cardiomyopathy in dystrophin-deficient hearts","volume":"5","author":"Hammers","year":"2016","journal-title":"J. Am. Heart Assoc."},{"key":"B70","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1038\/nm0302-253","article-title":"Modular flexibility of dystrophin: Implications for gene therapy of duchenne muscular dystrophy","volume":"8","author":"Harper","year":"2002","journal-title":"Nat. Med."},{"key":"B71","doi-asserted-by":"publisher","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: The protein product of the duchenne muscular dystrophy locus","volume":"51","author":"Hoffman","year":"1987","journal-title":"Cell"},{"key":"B72","doi-asserted-by":"publisher","first-page":"43","DOI":"10.1016\/j.steroids.2018.02.010","article-title":"Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes","volume":"134","author":"Hoffman","year":"2018","journal-title":"Steroids"},{"key":"B73","doi-asserted-by":"publisher","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":"Hoffman","year":"2019","journal-title":"Neurology"},{"key":"B74","doi-asserted-by":"publisher","first-page":"164","DOI":"10.1002\/1531-8249(200008)48:2<164::aid-ana5>3.0.co;2-b","article-title":"Sequence specificity of aminoglycoside-induced stop codon readthrough: Potential implications for treatment of Duchenne muscular dystrophy","volume":"48","author":"Howard","year":"2000","journal-title":"Ann. Neurol."},{"key":"B75","volume-title":"Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim results from the phase 3 essence trial (Poster# 49).","author":"Iannaccone","year":"2022"},{"key":"B76","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1007\/s10863-014-9571-y","article-title":"Idebenone and neuroprotection: Antioxidant, pro-oxidant, or electron carrier?","volume":"47","author":"Jaber","year":"2015","journal-title":"J. Bioenerg. Biomembr."},{"key":"B77","doi-asserted-by":"publisher","first-page":"S95","DOI":"10.1016\/j.nmd.2016.06.037","article-title":"BMS-986089: A novel adnectin protein that dose dependently lowers free myostatin and increases muscle volume and lean body mass","volume":"26","author":"Jacobsen","year":"2016","journal-title":"Neuromuscul. Disord."},{"key":"B78","article-title":"Sarepta therapeutics announces that at the 23rd international congress of the world muscle SocietyPresented positive updated results from the four children dosed in the gene therapy micro-dystrophin trial to treat patients with duchenne muscular dystrophy","author":"Jerry Mendell","year":"2018","journal-title":"Sarepta Ther."},{"key":"B79","article-title":"PF-06939926 continues to show safety and efficacy in duchenne muscular dystrophy","author":"Johnson","year":"2021","journal-title":"NeurologyLive"},{"key":"B80","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1016\/j.nmd.2011.08.001","article-title":"Endpoint measures in the mdx mouse relevant for muscular dystrophy pre-clinical studies","volume":"22","author":"Kobayashi","year":"2012","journal-title":"Neuromuscul. Disord."},{"key":"B81","doi-asserted-by":"publisher","first-page":"511","DOI":"10.1038\/nature07414","article-title":"Sarcolemma-localized nNOS is required to maintain activity after mild exercise","volume":"456","author":"Kobayashi","year":"2008","journal-title":"Nature"},{"key":"B82","doi-asserted-by":"publisher","first-page":"eaan0713","DOI":"10.1126\/scitranslmed.aan0713","article-title":"Systemic administration of the antisense oligonucleotide NS-065\/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy","volume":"10","author":"Komaki","year":"2018","journal-title":"Sci. Transl. Med."},{"key":"B83","doi-asserted-by":"publisher","first-page":"2393","DOI":"10.1002\/acn3.51235","article-title":"Viltolarsen in Japanese duchenne muscular dystrophy patients: A phase 1\/2 study","volume":"7","author":"Komaki","year":"2020","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"B84","doi-asserted-by":"publisher","first-page":"956","DOI":"10.1126\/science.8052854","article-title":"Inhibition of NF-kappa B by sodium salicylate and aspirin","volume":"265","author":"Kopp","year":"1994","journal-title":"Science"},{"key":"B85","doi-asserted-by":"publisher","first-page":"16105","DOI":"10.1038\/ncomms16105","article-title":"Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy","volume":"8","author":"Le Guiner","year":"2017","journal-title":"Nat. Commun."},{"key":"B86","doi-asserted-by":"publisher","first-page":"541","DOI":"10.1002\/ana.24214","article-title":"Sildenafil does not improve cardiomyopathy in Duchenne\/Becker muscular dystrophy","volume":"76","author":"Leung","year":"2014","journal-title":"Ann. Neurol."},{"key":"B87","doi-asserted-by":"publisher","first-page":"318","DOI":"10.1002\/psp4.12753","article-title":"Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy","volume":"11","author":"Lingineni","year":"2022","journal-title":"CPT. Pharmacometrics Syst. Pharmacol."},{"key":"B88","doi-asserted-by":"publisher","first-page":"468","DOI":"10.1183\/09031936.06.00059906","article-title":"Ventilatory assistance in patients with Duchenne muscular dystrophy","volume":"28","author":"Lofaso","year":"2006","journal-title":"Eur. Respir. J."},{"key":"B89","doi-asserted-by":"publisher","first-page":"55","DOI":"10.1038\/339055a0","article-title":"An autosomal transcript in skeletal muscle with homology to dystrophin","volume":"339","author":"Love","year":"1989","journal-title":"Nature"},{"key":"B90","doi-asserted-by":"publisher","first-page":"822389","DOI":"10.3389\/fimmu.2021.822389","article-title":"Editorial: \"AAV gene therapy: Immunology and immunotherapeutics","volume":"12","author":"Martinez-Navio","year":"2021","journal-title":"Front. Immunol."},{"key":"B91","doi-asserted-by":"publisher","first-page":"CD003725","DOI":"10.1002\/14651858.CD003725.pub4","article-title":"Corticosteroids for the treatment of Duchenne muscular dystrophy","volume":"5","author":"Matthews","year":"2016","journal-title":"Cochrane Database Syst. Rev."},{"key":"B92","doi-asserted-by":"publisher","first-page":"1489","DOI":"10.1016\/S0140-6736(17)31611-2","article-title":"Ataluren in patients with nonsense mutation duchenne muscular dystrophy (ACT DMD): A multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"390","author":"McDonald","year":"2017","journal-title":"Lancet"},{"key":"B93","doi-asserted-by":"publisher","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":"McDonald","year":"2018","journal-title":"Lancet"},{"key":"B94","doi-asserted-by":"publisher","first-page":"1049","DOI":"10.1016\/S0140-6736(22)00012-5","article-title":"Repeated intravenous cardiosphere-derived cell therapy in late-stage duchenne muscular dystrophy (HOPE-2): A multicentre, randomised, double-blind, placebo-controlled, phase 2 trial","volume":"399","author":"McDonald","year":"2022","journal-title":"Lancet"},{"key":"B95","doi-asserted-by":"publisher","first-page":"989","DOI":"10.3233\/JND-210643","article-title":"Open-label evaluation of eteplirsen in patients with duchenne muscular dystrophy amenable to exon 51 skipping: PROMOVI trial","volume":"8","author":"McDonald","year":"2021","journal-title":"J. Neuromuscul. Dis."},{"key":"B96","doi-asserted-by":"publisher","first-page":"e5","DOI":"10.1155\/2013\/406163","article-title":"Twenty-four hour noninvasive ventilation in duchenne muscular dystrophy: A safe alternative to tracheostomy","volume":"20","author":"McKim","year":"2013","journal-title":"Can. Respir. J."},{"key":"B97","doi-asserted-by":"publisher","first-page":"199","DOI":"10.1001\/jamacardio.2016.4910","article-title":"Cardiomyopathy in muscular dystrophy: When to treat?","volume":"2","author":"McNally","year":"2017","journal-title":"JAMA Cardiol."},{"key":"B98","doi-asserted-by":"publisher","first-page":"75","DOI":"10.1146\/annurev.med.58.011706.144703","article-title":"New approaches in the therapy of cardiomyopathy in muscular dystrophy","volume":"58","author":"McNally","year":"2007","journal-title":"Annu. Rev. Med."},{"key":"B99","doi-asserted-by":"publisher","first-page":"83","DOI":"10.1038\/387083a0","article-title":"Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member","volume":"387","author":"McPherron","year":"1997","journal-title":"Nature"},{"key":"B100","doi-asserted-by":"publisher","first-page":"12457","DOI":"10.1073\/pnas.94.23.12457","article-title":"Double muscling in cattle due to mutations in the myostatin gene","volume":"94","author":"McPherron","year":"1997","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B101","doi-asserted-by":"publisher","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy","volume":"74","author":"Mendell","year":"2013","journal-title":"Ann. Neurol."},{"key":"B102","doi-asserted-by":"publisher","first-page":"1122","DOI":"10.1001\/jamaneurol.2020.1484","article-title":"Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with duchenne muscular dystrophy: A nonrandomized controlled trial","volume":"77","author":"Mendell","year":"2020","journal-title":"JAMA Neurol."},{"key":"B103","doi-asserted-by":"publisher","first-page":"304","DOI":"10.1002\/ana.23528","article-title":"Evidence-based path to newborn screening for Duchenne muscular dystrophy","volume":"71","author":"Mendell","year":"2012","journal-title":"Ann. Neurol."},{"key":"B104","doi-asserted-by":"publisher","first-page":"576","DOI":"10.1016\/j.nmd.2016.05.016","article-title":"Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy","volume":"26","author":"Mercuri","year":"2016","journal-title":"Neuromuscul. Disord."},{"key":"B105","doi-asserted-by":"publisher","first-page":"2745","DOI":"10.2147\/DDDT.S110163","article-title":"Anti-inflammatory drugs for duchenne muscular dystrophy: Focus on skeletal muscle-releasing factors","volume":"10","author":"Miyatake","year":"2016","journal-title":"Drug Des. devel. Ther."},{"key":"B106","doi-asserted-by":"publisher","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":"Moat","year":"2013","journal-title":"Eur. J. Hum. Genet."},{"key":"B107","volume-title":"Safety and tolerability of PF-06939926 in ambulatory boys with Duchenne muscular dystrophy: A phase 1b multicenter, OpenLabel, dose ascending study","author":"Moorehead","year":"2020"},{"key":"B108","doi-asserted-by":"publisher","first-page":"e79","DOI":"10.1371\/journal.pgen.0030079","article-title":"A mutation in the myostatin gene increases muscle mass and enhances racing performance in heterozygote dogs","volume":"3","author":"Mosher","year":"2007","journal-title":"PLoS Genet."},{"key":"B109","doi-asserted-by":"publisher","first-page":"804","DOI":"10.1038\/d41573-021-00164-x","article-title":"Gene therapy community grapples with toxicity issues, as pipeline matures","volume":"20","author":"Mullard","year":"2021","journal-title":"Nat. Rev. Drug Discov."},{"key":"B110","doi-asserted-by":"publisher","first-page":"922","DOI":"10.1002\/cpdd.642","article-title":"A phase 1b trial to assess the pharmacokinetics of ezutromid in pediatric duchenne muscular dystrophy patients on a balanced diet","volume":"8","author":"Muntoni","year":"2019","journal-title":"Clin. Pharmacol. Drug Dev."},{"key":"B111","doi-asserted-by":"publisher","first-page":"2425","DOI":"10.2353\/ajpath.2010.090932","article-title":"Antibody-directed myostatin inhibition improves diaphragm pathology in young but not adult dystrophic mdx mice","volume":"176","author":"Murphy","year":"2010","journal-title":"Am. J. Pathol."},{"key":"B112","doi-asserted-by":"publisher","first-page":"2085","DOI":"10.1212\/WNL.0000000000000498","article-title":"PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy","volume":"82","author":"Nelson","year":"2014","journal-title":"Neurology"},{"key":"B113","doi-asserted-by":"publisher","first-page":"913","DOI":"10.1016\/j.nmd.2007.07.005","article-title":"Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human","volume":"17","author":"Neri","year":"2007","journal-title":"Neuromuscul. Disord."},{"key":"B114","doi-asserted-by":"publisher","first-page":"799","DOI":"10.1124\/mol.107.038794","article-title":"Separating transrepression and transactivation: A distressing divorce for the glucocorticoid receptor?","volume":"72","author":"Newton","year":"2007","journal-title":"Mol. Pharmacol."},{"key":"B115","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1002\/path.4054","article-title":"Sildenafil reduces respiratory muscle weakness and fibrosis in the mdx mouse model of Duchenne muscular dystrophy","volume":"228","author":"Percival","year":"2012","journal-title":"J. Pathol."},{"key":"B116","article-title":"A message from pfizer on our DMD clinical program","year":"","journal-title":"Pfizer Inc"},{"key":"B117","article-title":"Pfizer receives FDA fast track designation for duchenne muscular dystrophy investigational gene therapy","year":"","journal-title":"Pfizer Inc"},{"key":"B118","article-title":"Pfizer terminates domagrozumab (PF-06252616) clinical studies for the treatment of duchenne muscular dystrophy","year":"2018","journal-title":"Pfizer Inc"},{"key":"B119","article-title":"Pfizer\u2019s new phase 1b results of gene therapy in ambulatory boys with duchenne muscular dystrophy (DMD) support advancement into pivotal phase 3 study","year":"","journal-title":"Pfizer Inc"},{"key":"B120","article-title":"Statement from the pfizer DMD gene therapy team","year":"","journal-title":"Pfizer Inc"},{"key":"B121","doi-asserted-by":"publisher","first-page":"573","DOI":"10.1089\/hum.2022.29211.bfs","article-title":"Food and drug administration lifts clinical hold on pfizer duchenne muscular dystrophy gene therapy linked to patient death","volume":"33","author":"Philippidis","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"B122","article-title":"CHMP adopts positive opinion for the expansion of the Translarna\u2122 (ataluren) label to include patients as young as 2 Years of age","year":"2018","journal-title":"PTC Ther"},{"key":"B123","doi-asserted-by":"publisher","first-page":"582","DOI":"10.1161\/CIRCULATIONAHA.111.031716","article-title":"Early treatment with lisinopril and spironolactone preserves cardiac and skeletal muscle in Duchenne muscular dystrophy mice","volume":"124","author":"Rafael-Fortney","year":"2011","journal-title":"Circulation"},{"key":"B124","doi-asserted-by":"publisher","first-page":"e013501","DOI":"10.1161\/JAHA.119.013501","article-title":"Stabilization of early duchenne cardiomyopathy with aldosterone inhibition: Results of the multicenter AIDMD trial","volume":"8","author":"Raman","year":"2019","journal-title":"J. Am. Heart Assoc."},{"key":"B125","doi-asserted-by":"publisher","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":"Raman","year":"2015","journal-title":"Lancet. Neurol."},{"key":"B126","doi-asserted-by":"publisher","first-page":"7230","DOI":"10.1128\/MCB.23.20.7230-7242.2003","article-title":"Myostatin signals through a transforming growth factor beta-like signaling pathway to block adipogenesis","volume":"23","author":"Rebbapragada","year":"2003","journal-title":"Mol. Cell. Biol."},{"key":"B127","doi-asserted-by":"publisher","first-page":"25","DOI":"10.1016\/S2213-2600(19)30262-0","article-title":"Pamrevlumab, an anti-connective tissue growth factor therapy, for idiopathic pulmonary fibrosis (PRAISE): A phase 2, randomised, double-blind, placebo-controlled trial","volume":"8","author":"Richeldi","year":"2020","journal-title":"Lancet. Respir. Med."},{"key":"B128","doi-asserted-by":"publisher","first-page":"e0152840","DOI":"10.1371\/journal.pone.0152840","article-title":"Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to pediatric patients with duchenne muscular dystrophy","volume":"11","author":"Ricotti","year":"2016","journal-title":"PLoS One"},{"key":"B129","article-title":"Roche Genentech releases letter to DMD community announcing discontinuation of clinical trials program for DMD","year":"2019","journal-title":"Roche Genentech"},{"key":"B130","doi-asserted-by":"publisher","first-page":"13818","DOI":"10.1073\/pnas.250379497","article-title":"Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy","volume":"97","author":"Sander","year":"2000","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B131","article-title":"Santhera and ReveraGen announce new 2.5-year treatment data with vamorolone in duchenne muscular dystrophy","year":"","journal-title":"Santhera Pharm"},{"key":"B132","article-title":"Santhera and ReveraGen announce positive topline results with vamorolone after completion of the VISION-DMD study","year":"","journal-title":"Santhera Pharm"},{"key":"B133","article-title":"Santhera and ReveraGen to present findings from pivotal VISION-DMD study with vamorolone at parent Project muscular dystrophy 2021 conference","year":"","journal-title":"Santhera Pharm"},{"key":"B134","article-title":"Santhera receives negative CHMP opinion on appeal for authorization of Raxone\u00ae in duchenne muscular dystrophy","year":"2018","journal-title":"Santhera Pharm"},{"key":"B135","article-title":"Santhera to discontinue phase 3 SIDEROS study and development of Puldysa\u00ae in duchenne muscular dystrophy (DMD) and focus on vamorolone","year":"2020","journal-title":"Santhera Pharm"},{"key":"B136","article-title":"Santhera updates on U.S. Regulatory filing for Raxone\u00ae (idebenone) in duchenne muscular dystrophy (DMD)","year":"2016","journal-title":"Santhera Pharm"},{"key":"B137","article-title":"Sarepta therapeutics announces positive expression results from the casimersen (SRP-4045) arm of the ESSENCE study","year":"2019","journal-title":"Sarepta Ther"},{"key":"B138","volume-title":"Sarepta therapeutics announces top-line results for Part 1 of study 102 evaluating SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy.","year":""},{"key":"B139","article-title":"Sarepta therapeutics\u2019 gene therapy SRP-9001 shows statistically significant functional improvements compared to pre-specified matched external control in Part 2 of study SRP-9001-102 for the treatment of duchenne muscular dystrophy","year":"2022","journal-title":"Sarepta Ther."},{"key":"B140","article-title":"Sarepta therapeutics\u2019 investigational gene therapy for the treatment of Duchenne muscular dystrophy, SRP-9001, demonstrates robust expression and consistent safety profile using sarepta\u2019s commercial process material","year":"","journal-title":"Sarepta therapeutics"},{"key":"B141","article-title":"Sarepta therapeutics receives fast track designation for SRP-9001 micro-dystrophin gene therapy for the treatment of duchenne muscular dystrophy","year":"2020","journal-title":"Sarepta Ther"},{"key":"B142","article-title":"Sarepta therapeutics reports positive clinical results from phase 2 MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51","year":"","journal-title":"Sarepta therapeutics"},{"key":"B143","doi-asserted-by":"publisher","first-page":"23","DOI":"10.1016\/s0163-7258(02)00297-8","article-title":"Mechanisms involved in the side effects of glucocorticoids","volume":"96","author":"Schacke","year":"2002","journal-title":"Pharmacol. Ther."},{"key":"B144","volume-title":"SGT-001 microdystrophin gene therapy for Duchenne muscular dystrophy","author":"Schneider","year":"2017"},{"key":"B145","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1002\/aja.1001980403","article-title":"Expression of the dystrophin-related protein (utrophin) gene during mouse embryogenesis","volume":"198","author":"Schofield","year":"1993","journal-title":"Dev. Dyn."},{"key":"B146","doi-asserted-by":"publisher","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":"Schram","year":"2013","journal-title":"J. Am. Coll. Cardiol."},{"key":"B147","doi-asserted-by":"publisher","first-page":"2682","DOI":"10.1056\/NEJMoa040933","article-title":"Myostatin mutation associated with gross muscle hypertrophy in a child","volume":"350","author":"Schuelke","year":"2004","journal-title":"N. Engl. J. Med."},{"key":"B148","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1089\/nat.2021.0043","article-title":"Long-term safety and efficacy data of golodirsen in ambulatory patients with duchenne muscular dystrophy amenable to exon 53 skipping: A first-in-human, multicenter, two-part, open-label, phase 1\/2 trial","volume":"32","author":"Servais","year":"2022","journal-title":"Nucleic Acid. Ther."},{"key":"B149","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1007\/s00204-021-03184-z","article-title":"Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: An update","volume":"96","author":"Sheikh","year":"2022","journal-title":"Arch. Toxicol."},{"key":"B150","doi-asserted-by":"publisher","first-page":"750","DOI":"10.1038\/mt.2012.283","article-title":"Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy","volume":"21","author":"Shin","year":"2013","journal-title":"Mol. Ther."},{"key":"B151","doi-asserted-by":"publisher","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":"Smith","year":"2020","journal-title":"PLoS Med."},{"key":"B152","article-title":"Solid Biosciences announces clinical hold on SGT-001 phase I\/II clinical trial for duchenne muscular dystrophy","year":"","journal-title":"Solid Biosci."},{"key":"B153","article-title":"Solid Biosciences announces FDA lifts clinical hold on IGNITE DMD clinical trial","year":"2020","journal-title":"Solid Biosci"},{"key":"B154","article-title":"Solid Biosciences announces FDA removes clinical hold on SGT-001","year":"","journal-title":"Solid Biosci."},{"key":"B155","article-title":"Solid Biosciences announces preliminary SGT-001 data and intention to dose escalate in IGNITE DMD clinical trial for Duchenne muscular dystrophy","year":"","journal-title":"Solid Biosciences"},{"key":"B156","article-title":"Solid Biosciences outlines its strategic priorities for 2022 and announces corporate updates","year":"2022","journal-title":"Solid Biosciences"},{"key":"B157","article-title":"Solid Biosciences provides SGT-001 program update","year":"","journal-title":"Solid Biosci"},{"key":"B158","article-title":"Solid Biosciences reports 1.5-year data from patients in the ongoing IGNITE DMD phase I\/II clinical trial of SGT-001","year":"2021","journal-title":"Solid Biosci."},{"key":"B159","article-title":"Summit announces new analysis showing ezutromid significantly reduced muscle inflammation in phase 2 clinical trial in DMD","year":"","journal-title":"Summit Ther."},{"key":"B160","article-title":"Summit therapeutics discontinues ezutromid for DMD after disappointing phase 2 trial results","year":"","journal-title":"Summit Ther."},{"key":"B161","doi-asserted-by":"publisher","first-page":"e866","DOI":"10.1212\/WNL.0000000000006950","article-title":"Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial","volume":"92","author":"Taylor","year":"2019","journal-title":"Neurology"},{"key":"B162","doi-asserted-by":"publisher","first-page":"1441","DOI":"10.1038\/4033","article-title":"Expression of full-length utrophin prevents muscular dystrophy in mdx mice","volume":"4","author":"Tinsley","year":"1998","journal-title":"Nat. Med."},{"key":"B163","doi-asserted-by":"publisher","first-page":"304","DOI":"10.3389\/fneur.2020.00304","article-title":"Longitudinal study of three microRNAs in duchenne muscular dystrophy and becker muscular dystrophy","volume":"11","author":"Trifunov","year":"2020","journal-title":"Front. Neurol."},{"key":"B164","doi-asserted-by":"publisher","first-page":"e31937","DOI":"10.1371\/journal.pone.0031937","article-title":"The effects of low levels of dystrophin on mouse muscle function and pathology","volume":"7","author":"van Putten","year":"2012","journal-title":"PLoS One"},{"key":"B165","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1016\/j.tem.2017.10.010","article-title":"Therapeutic mechanisms of glucocorticoids","volume":"29","author":"Vandewalle","year":"2018","journal-title":"Trends Endocrinol. Metab."},{"key":"B166","doi-asserted-by":"publisher","first-page":"1811","DOI":"10.1212\/WNL.0000000000004570","article-title":"A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy","volume":"89","author":"Victor","year":"2017","journal-title":"Neurology"},{"key":"B167","doi-asserted-by":"publisher","first-page":"1217","DOI":"10.1021\/acs.jmedchem.5b01961","article-title":"Synthesis and characterization of fatty acid conjugates of niacin and salicylic acid","volume":"59","author":"Vu","year":"2016","journal-title":"J. Med. Chem."},{"key":"B168","doi-asserted-by":"publisher","first-page":"2120","DOI":"10.1038\/mt.2012.181","article-title":"Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping","volume":"20","author":"Vulin","year":"2012","journal-title":"Mol. Ther."},{"key":"B169","doi-asserted-by":"publisher","first-page":"827","DOI":"10.1016\/j.nmd.2015.08.005","article-title":"The first exon duplication mouse model of duchenne muscular dystrophy: A tool for therapeutic development","volume":"25","author":"Vulin","year":"2015","journal-title":"Neuromuscul. Disord."},{"key":"B170","doi-asserted-by":"publisher","first-page":"2020167","DOI":"10.1016\/j.nmd.2021.01.001","article-title":"Corrigendum to \u201cRandomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy\u201d [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502]","volume":"30","author":"Wagner","year":"","journal-title":"Neuromuscul. Disord."},{"key":"B171","doi-asserted-by":"publisher","first-page":"492","DOI":"10.1016\/j.nmd.2020.05.002","article-title":"Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy","volume":"30","author":"Wagner","year":"2020","journal-title":"Neuromuscul. Disord."},{"key":"B172","doi-asserted-by":"publisher","first-page":"285","DOI":"10.1002\/mus.27347","article-title":"Safety, tolerability, and pharmacokinetics of casimersen in patients with duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial","volume":"64","author":"Wagner","year":"","journal-title":"Muscle Nerve"},{"key":"B173","article-title":"A phase 1b\/2 study of the anti-myostatin adnectin RG6206 (BMS-986089) in ambulatory boys with duchenne muscular dystrophy (DMD): A 72-week treatment update","author":"Wagner","year":"2019","journal-title":"Am. Acad. Neurosci. Annu. Meet."},{"key":"B174","doi-asserted-by":"publisher","first-page":"13714","DOI":"10.1073\/pnas.240335297","article-title":"Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model","volume":"97","author":"Wang","year":"2000","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"B175","doi-asserted-by":"publisher","first-page":"e97957","DOI":"10.1371\/journal.pone.0097957","article-title":"Omega-3 free fatty acids suppress macrophage inflammasome activation by inhibiting NF-\u03baB activation and enhancing autophagy.","volume":"9","author":"Williams-Bey","year":"2014","journal-title":"PLoS One"},{"key":"B176","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1038\/23948","article-title":"The anti-inflammatory agents aspirin and salicylate inhibit the activity of I(kappa)B kinase-beta","volume":"396","author":"Yin","year":"1998","journal-title":"Nature"},{"key":"B177","doi-asserted-by":"publisher","first-page":"788","DOI":"10.3389\/fphys.2017.00788","article-title":"mTOR as a key regulator in maintaining skeletal muscle mass","volume":"8","author":"Yoon","year":"2017","journal-title":"Front. Physiol."},{"key":"B178","doi-asserted-by":"publisher","first-page":"174","DOI":"10.1097\/MNH.0b013e3282f56854","article-title":"Mechanisms of mineralocorticoid receptor-mediated cardiac fibrosis and vascular inflammation","volume":"17","author":"Young","year":"2008","journal-title":"Curr. Opin. Nephrol. Hypertens."},{"key":"B179","doi-asserted-by":"publisher","first-page":"188","DOI":"10.1186\/s13023-021-01837-x","article-title":"Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy","volume":"16","author":"Zhang","year":"2021","journal-title":"Orphanet J. Rare Dis."},{"key":"B180","doi-asserted-by":"publisher","first-page":"1049","DOI":"10.1359\/jbmr.091041","article-title":"Capacity of omega-3 fatty acids or eicosapentaenoic acid to counteract weightlessness-induced bone loss by inhibiting NF-kappaB activation: From cells to bed rest to astronauts","volume":"25","author":"Zwart","year":"2010","journal-title":"J. Bone Min. Res."}],"container-title":["Frontiers in Pharmacology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fphar.2022.950651\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,7,22]],"date-time":"2022-07-22T06:11:06Z","timestamp":1658470266000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fphar.2022.950651\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,7,22]]},"references-count":180,"alternative-id":["10.3389\/fphar.2022.950651"],"URL":"https:\/\/doi.org\/10.3389\/fphar.2022.950651","relation":{},"ISSN":["1663-9812"],"issn-type":[{"value":"1663-9812","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,7,22]]},"article-number":"950651"}}