{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T15:03:07Z","timestamp":1793718187642,"version":"4.6.1"},"reference-count":104,"publisher":"MDPI AG","issue":"4","license":[{"start":{"date-parts":[[2024,7,5]],"date-time":"2024-07-05T00:00:00Z","timestamp":1720137600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Neurology International"],"abstract":"<jats:p>Significant progress has been achieved in understanding Duchenne muscular dystrophy (DMD) mechanisms and developing treatments to slow disease progression. This review article thoroughly assesses primary and secondary DMD therapies, focusing on innovative modalities. The primary therapy addresses the genetic abnormality causing DMD, specifically the absence or reduced expression of dystrophin. Gene replacement therapies, such as exon skipping, readthrough, and gene editing technologies, show promise in restoring dystrophin expression. Adeno-associated viruses (AAVs), a recent advancement in viral vector-based gene therapies, have shown encouraging results in preclinical and clinical studies. Secondary therapies aim to maintain muscle function and improve quality of life by mitigating DMD symptoms and complications. Glucocorticoid drugs like prednisone and deflazacort have proven effective in slowing disease progression and delaying loss of ambulation. Supportive treatments targeting calcium dysregulation, histone deacetylase, and redox imbalance are also crucial for preserving overall health and function. Additionally, the review includes a detailed table of ongoing and approved clinical trials for DMD, exploring various therapeutic approaches such as gene therapies, exon skipping drugs, utrophin modulators, anti-inflammatory agents, and novel compounds. This highlights the dynamic research field and ongoing efforts to develop effective DMD treatments.<\/jats:p>","DOI":"10.3390\/neurolint16040055","type":"journal-article","created":{"date-parts":[[2024,7,9]],"date-time":"2024-07-09T15:27:20Z","timestamp":1720538840000},"page":"731-760","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Molecular and Biochemical Therapeutic Strategies for Duchenne Muscular Dystrophy"],"prefix":"10.3390","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0009-0000-7427-9786","authenticated-orcid":false,"given":"Lakshmi","family":"Krishna","sequence":"first","affiliation":[{"name":"Department of Biochemistry, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0383-2366","authenticated-orcid":false,"given":"Akila","family":"Prashant","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"},{"name":"Department of Medical Genetics, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yogish H.","family":"Kumar","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutical Chemistry, JSS College of Pharmacy, Mysuru, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5095-8602","authenticated-orcid":false,"given":"Shasthara","family":"Paneyala","sequence":"additional","affiliation":[{"name":"Department of Neurology, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siddaramappa J.","family":"Patil","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Narayana Hrudalaya Health Hospital\/Mazumdar Shah, Bengaluru 560099, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6744-8430","authenticated-orcid":false,"given":"Shobha Chikkavaddaragudi","family":"Ramachandra","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prashant","family":"Vishwanath","sequence":"additional","affiliation":[{"name":"Department of Biochemistry, JSS Medical College, JSS Academy of Higher Education & Research, Mysuru 570015, Karnataka, India"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,7,5]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"18","author":"Duan","year":"2021","journal-title":"Nat. Rev. Dis. Primers"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"421","DOI":"10.1161\/CIRCGEN.123.004221","article-title":"DMD-Associated Dilated Cardiomyopathy: Genotypes, Phenotypes, and Phenocopies","volume":"16","author":"Johnson","year":"2023","journal-title":"Circ. Genom. Precis. Med."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"1813","DOI":"10.1007\/s00424-021-02623-1","article-title":"Complexity of skeletal muscle degeneration: Multi-systems pathophysiology and organ crosstalk in dystrophinopathy","volume":"473","author":"Ohlendieck","year":"2021","journal-title":"Pflug. Arch."},{"key":"ref_4","doi-asserted-by":"crossref","unstructured":"Deepha, S., Vengalil, S., Preethish-Kumar, V., Polavarapu, K., Nalini, A., Gayathri, N., and Purushottam, M. (2017). MLPA identification of dystrophin mutations and in silico evaluation of the predicted protein in dystrophinopathy cases from India. BMC Med. Genet., 18.","DOI":"10.1186\/s12881-017-0431-6"},{"key":"ref_5","doi-asserted-by":"crossref","unstructured":"Bovolenta, M., Neri, M., Fini, S., Fabris, M., Trabanelli, C., Venturoli, A., EMartoni, E., Bassi, E., Spitali, P., and Brioschi, S. (2008). A novel custom high density-comparative genomic hybridization array detects common rearrangements as well as deep intronic mutations in dystrophinopathies. BMC Genom., 9.","DOI":"10.1186\/1471-2164-9-572"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"370","DOI":"10.1080\/00207454.2020.1740218","article-title":"Common therapeutic advances for Duchenne muscular dystrophy (DMD)","volume":"131","author":"Salmaninejad","year":"2021","journal-title":"Int. J. Neurosci."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"854","DOI":"10.1080\/00207454.2018.1430694","article-title":"Duchenne muscular dystrophy: An updated review of common available therapies","volume":"128","author":"Salmaninejad","year":"2018","journal-title":"Int. J. Neurosci."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"139","DOI":"10.2174\/156652312800840603","article-title":"Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors","volume":"12","author":"Seto","year":"2012","journal-title":"Curr. Gene Ther."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.gendis.2019.12.007","article-title":"Promising therapeutic approaches using CRISPR\/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy","volume":"8","author":"Mollanoori","year":"2021","journal-title":"Genes Dis."},{"key":"ref_10","doi-asserted-by":"crossref","unstructured":"O\u2019Leary, D.A., Sharif, O., Anderson, P., Tu, B., Welch, G., Zhou, Y., Caldwell, J., Engels, I., and Brinker, A. (2009). Identification of Small Molecule and Genetic Modulators of AON-Induced Dystrophin Exon Skipping by High-Throughput Screening. PLoS ONE, 4.","DOI":"10.1371\/journal.pone.0008348"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"S343","DOI":"10.3233\/JND-210682","article-title":"Exon-Skipping in Duchenne Muscular Dystrophy","volume":"8","author":"Takeda","year":"2021","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"1604","DOI":"10.1016\/j.ajpath.2022.07.016","article-title":"Dystrophin Restoration after Adeno-Associated Virus U7\u2013Mediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model","volume":"192","author":"Monceau","year":"2022","journal-title":"Am. J. Pathol."},{"key":"ref_13","doi-asserted-by":"crossref","unstructured":"Sun, C., Shen, L., Zhang, Z., and Xie, X. (2020). Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update. Genes, 11.","DOI":"10.3390\/genes11080837"},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"18168","DOI":"10.3390\/molecules201018168","article-title":"Duchenne Muscular Dystrophy: From Diagnosis to Therapy","volume":"20","author":"Falzarano","year":"2015","journal-title":"Molecules"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1089\/nat.2018.0756","article-title":"A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe","volume":"29","author":"Goemans","year":"2019","journal-title":"Nucleic Acid. Ther."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1080\/14712598.2017.1271872","article-title":"Exon skipping: A first in class strategy for Duchenne muscular dystrophy","volume":"17","author":"Niks","year":"2017","journal-title":"Expert Opin. Biol. Ther."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1007\/s40265-021-01512-2","article-title":"Casimersen: First Approval","volume":"81","author":"Shirley","year":"2021","journal-title":"Drugs"},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"588","DOI":"10.1097\/WCO.0b013e328357b0be","article-title":"Gene therapy for Duchenne muscular dystrophy","volume":"25","author":"Verhaart","year":"2012","journal-title":"Curr. Opin. Neurol."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"1249","DOI":"10.1038\/sj.gt.3302984","article-title":"Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products","volume":"14","author":"Yuasa","year":"2007","journal-title":"Gene Ther."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"840","DOI":"10.1007\/s13311-018-00687-z","article-title":"Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy","volume":"15","author":"Shieh","year":"2018","journal-title":"Neurotherapeutics"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1038\/s41392-021-00487-6","article-title":"Viral vector platforms within the gene therapy landscape","volume":"6","author":"Bulcha","year":"2021","journal-title":"Signal Transduct. Target. Ther."},{"key":"ref_22","doi-asserted-by":"crossref","unstructured":"Manini, A., Abati, E., Nuredini, A., Corti, S., and Comi, G.P. (2022). Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence. Front. Neurol., 12.","DOI":"10.3389\/fneur.2021.814174"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1080\/14712598.2020.1725469","article-title":"Clinical development on the frontier: Gene therapy for Duchenne muscular dystrophy","volume":"20","author":"Asher","year":"2020","journal-title":"Expert Opin. Biol. Ther."},{"key":"ref_24","doi-asserted-by":"crossref","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":"ref_25","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.1016\/j.ymthe.2022.03.004","article-title":"Durability of transgene expression after rAAV gene therapy","volume":"30","author":"Muhuri","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"3087","DOI":"10.1016\/j.yexcr.2010.05.022","article-title":"Gene therapy for muscle disease","volume":"316","author":"Takeda","year":"2010","journal-title":"Exp. Cell Res."},{"key":"ref_27","doi-asserted-by":"crossref","unstructured":"Yao, S., Chen, Z., Yu, Y., Zhang, N., Jiang, H., Zhang, G., Zhang, Z., and Zhang, B. (2021). Current Pharmacological Strategies for Duchenne Muscular Dystrophy. Front. Cell Dev. Biol., 9.","DOI":"10.3389\/fcell.2021.689533"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"1369","DOI":"10.1038\/s41431-021-00811-2","article-title":"Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy","volume":"29","author":"Davies","year":"2021","journal-title":"Eur. J. Hum. Genet."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"16007","DOI":"10.1038\/ncomms16007","article-title":"Correction: Corrigendum: Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy","volume":"8","author":"Bengtsson","year":"2017","journal-title":"Nat. Commun."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1038\/mt.2015.192","article-title":"CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice","volume":"24","author":"Xu","year":"2016","journal-title":"Mol. Ther."},{"key":"ref_32","doi-asserted-by":"crossref","unstructured":"Yang, H., Ren, S., Yu, S., Pan, H., Li, T., Ge, S., and Xia, N. (2020). Methods Favoring Homology-Directed Repair Choice in Response to CRISPR\/Cas9 Induced-Double Strand Breaks. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21186461"},{"key":"ref_33","first-page":"353","article-title":"Mechanism and Applications of CRISPR\/Cas-9-Mediated Genome Editing","volume":"15","author":"Mengstie","year":"2021","journal-title":"Biologics"},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"6244","DOI":"10.1038\/ncomms7244","article-title":"Multiplex CRISPR\/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy","volume":"6","author":"Ousterout","year":"2015","journal-title":"Nat. Commun."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"175675","DOI":"10.1016\/j.ejphar.2023.175675","article-title":"Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach","volume":"947","author":"Patterson","year":"2023","journal-title":"Eur. J. Pharmacol."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1080\/13543784.2021.1868434","article-title":"Developing DMD therapeutics: A review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies","volume":"30","author":"Sheikh","year":"2021","journal-title":"Expert Opin. Investig. Drugs"},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"830","DOI":"10.1038\/mt.2011.59","article-title":"Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD","volume":"19","author":"Pichavant","year":"2011","journal-title":"Mol. Ther."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"444","DOI":"10.3109\/10409238.2012.694846","article-title":"Suppression of premature termination codons as a therapeutic approach","volume":"47","author":"Keeling","year":"2012","journal-title":"Crit. Rev. Biochem. Mol. Biol."},{"key":"ref_39","first-page":"37","article-title":"Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy","volume":"6","author":"Bertoni","year":"2016","journal-title":"Degener. Neurol. Neuromuscul. Dis."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1002\/ana.1023","article-title":"Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations","volume":"49","author":"Wagner","year":"2001","journal-title":"Ann. Neurol."},{"key":"ref_41","first-page":"43","article-title":"Read-through approach for stop mutations in Duchenne muscular dystrophy","volume":"40","author":"Politano","year":"2021","journal-title":"Acta Myol."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"1158","DOI":"10.1177\/0883073810371129","article-title":"Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne\/Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)","volume":"25","author":"Finkel","year":"2010","journal-title":"J. Child Neurol."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"881","DOI":"10.1038\/mt.2009.322","article-title":"Combination of Myostatin Pathway Interference and Dystrophin Rescue Enhances Tetanic and Specific Force in Dystrophic mdx Mice","volume":"18","author":"Dumonceaux","year":"2010","journal-title":"Mol. Ther."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"245","DOI":"10.2174\/156652312800840577","article-title":"Interference with Myostatin\/ActRIIB Signaling as a Therapeutic Strategy for Duchenne Muscular Dystrophy","volume":"12","author":"Amthor","year":"2012","journal-title":"Curr. Gene Ther."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"1859","DOI":"10.1038\/s41467-017-01486-4","article-title":"Downregulation of myostatin pathway in neuromuscular diseases, explain challenges of anti-myostatin therapeutic approaches","volume":"8","author":"Mariot","year":"2017","journal-title":"Nat. Commun."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1111\/nan.12735","article-title":"Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies","volume":"47","year":"2021","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1016\/j.coph.2017.04.002","article-title":"Pharmacological advances for treatment in Duchenne muscular dystrophy","volume":"34","author":"Guiraud","year":"2017","journal-title":"Curr. Opin. Pharmacol."},{"key":"ref_48","doi-asserted-by":"crossref","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":"ref_49","doi-asserted-by":"crossref","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":"ref_50","doi-asserted-by":"crossref","unstructured":"Tinsley, J.M., Fairclough, R.J., Storer, R., Wilkes, F.J., Potter, A.C., Squire, S.E., Powell, A., Cozzoli, A., Capogrosso, R., and Lambert, A. (2011). Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse. PLoS ONE, 6.","DOI":"10.1371\/journal.pone.0019189"},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"4212","DOI":"10.1093\/hmg\/ddv154","article-title":"Second-generation compound for the modulation of utrophin in the therapy of DMD","volume":"24","author":"Guiraud","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"4640","DOI":"10.1093\/hmg\/ddp431","article-title":"Pharmacological activation of PPAR \/stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice","volume":"18","author":"Miura","year":"2009","journal-title":"Hum. Mol. Genet."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"C110","DOI":"10.1152\/ajpcell.00183.2011","article-title":"Chronic AMPK stimulation attenuates adaptive signaling in dystrophic skeletal muscle","volume":"302","author":"Ljubicic","year":"2012","journal-title":"Am. J. Physiol.-Cell Physiol."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"5090","DOI":"10.1096\/fj.201800081R","article-title":"Celecoxib treatment improves muscle function in mdx mice and increases utrophin A expression","volume":"32","author":"Adam","year":"2018","journal-title":"FASEB J."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1093\/hmg\/ddv444","article-title":"Combinatorial therapeutic activation with heparin and AICAR stimulates additive effects on utrophin A expression in dystrophic muscles","volume":"25","author":"Ahmed","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"ref_56","doi-asserted-by":"crossref","unstructured":"Mishra, M.K., Loro, E., Sengupta, K., Wilton, S.D., and Khurana, T.S. (2017). Functional improvement of dystrophic muscle by repression of utrophin: Let-7c interaction. PLoS ONE, 12.","DOI":"10.1371\/journal.pone.0182676"},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"1505","DOI":"10.1038\/s41591-019-0594-0","article-title":"Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models","volume":"25","author":"Song","year":"2019","journal-title":"Nat. Med."},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1007\/s11940-018-0513-6","article-title":"Current and Emerging Therapies for Duchenne Muscular Dystrophy","volume":"20","author":"Crone","year":"2018","journal-title":"Curr. Treat. Options Neurol."},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1096\/fj.12-215723","article-title":"Arginine butyrate: A therapeutic candidate for Duchenne muscular dystrophy","volume":"27","author":"Vianello","year":"2013","journal-title":"FASEB J."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"762","DOI":"10.1073\/pnas.1013067108","article-title":"Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice","volume":"108","author":"Amenta","year":"2011","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"ddw356","DOI":"10.1093\/hmg\/ddw356","article-title":"High levels of sarcospan are well tolerated and act as a sarcolemmal stabilizer to address skeletal muscle and pulmonary dysfunction in DMD","volume":"24","author":"Gibbs","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"ref_62","first-page":"5","article-title":"Thrombospondin expression in myofibers stabilizes muscle membranes","volume":"26","author":"Vanhoutte","year":"2016","journal-title":"elife"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"1227","DOI":"10.1007\/s43440-020-00134-x","article-title":"Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: Past, present, and future","volume":"72","author":"Dulak","year":"2020","journal-title":"Pharmacol. Rep."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"100877","DOI":"10.1016\/j.spen.2021.100877","article-title":"Therapeutic Approaches for Duchenne Muscular Dystrophy: Old and New","volume":"37","author":"Mackenzie","year":"2021","journal-title":"Semin. Pediatr. Neurol."},{"key":"ref_65","doi-asserted-by":"crossref","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":"2018","journal-title":"Lancet Neurol."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"463","DOI":"10.3233\/JND-210776","article-title":"Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy","volume":"9","author":"Biggar","year":"2022","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"39","DOI":"10.3233\/JND-200556","article-title":"Mechanisms and Clinical Applications of Glucocorticoid Steroids in Muscular Dystrophy","volume":"8","author":"Quattrocelli","year":"2021","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1212\/WNL.0000000000002337","article-title":"Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy","volume":"86","author":"Gloss","year":"2016","journal-title":"Neurology"},{"key":"ref_69","first-page":"CD003725","article-title":"Corticosteroids for the treatment of Duchenne muscular dystrophy","volume":"5","author":"Matthews","year":"2016","journal-title":"Cochrane Database Syst. Rev."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1002\/mus.26191","article-title":"Deflazacort versus prednisone\/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial","volume":"58","author":"Shieh","year":"2018","journal-title":"Muscle Nerve"},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"177","DOI":"10.2217\/cer-2019-0170","article-title":"Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: Experience at a single, large care center","volume":"9","author":"Marden","year":"2020","journal-title":"J. Comp. Eff. Res."},{"key":"ref_72","doi-asserted-by":"crossref","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":"ref_73","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":"Hoffman","year":"2019","journal-title":"Neurology"},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"e2144178","DOI":"10.1001\/jamanetworkopen.2021.44178","article-title":"Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy","volume":"5","author":"Mah","year":"2022","journal-title":"JAMA Netw. Open"},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"627","DOI":"10.1002\/jcph.842","article-title":"A Novel NF-\u03baB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients with Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects","volume":"57","author":"Donovan","year":"2017","journal-title":"J. Clin. Pharmacol."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"769","DOI":"10.3233\/JND-210689","article-title":"A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial","volume":"8","author":"Finkel","year":"2021","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_77","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 Move DMD phase 2 and open label extension trial","volume":"31","author":"Finkel","year":"2021","journal-title":"Neuromuscul. Disord."},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1586\/14779072.5.1.99","article-title":"Cardiomyopathy of Duchenne muscular dystrophy: Pathogenesis and prospect of membrane sealants as a new therapeutic approach","volume":"5","author":"Townsend","year":"2007","journal-title":"Expert Rev. Cardiovasc. Ther."},{"key":"ref_79","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.ijcard.2020.03.031","article-title":"Protective effects of rimeporide on left ventricular function in golden retriever muscular dystrophy dogs","volume":"312","author":"Ghaleh","year":"2020","journal-title":"Int. J. Cardiol."},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1038\/mt.2014.200","article-title":"A Phase 1\/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy","volume":"23","author":"Mendell","year":"2015","journal-title":"Mol. Ther."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1002\/prca.200900116","article-title":"Proteomic profile of differentially expressed plasma proteins from dystrophic mice and following suberoylanilide hydroxamic acid treatment","volume":"4","author":"Colussi","year":"2010","journal-title":"Proteom. Clin. Appl."},{"key":"ref_82","doi-asserted-by":"crossref","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":"ref_83","doi-asserted-by":"crossref","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":"ref_84","doi-asserted-by":"crossref","first-page":"174","DOI":"10.1002\/mus.22047","article-title":"CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy","volume":"44","author":"Spurney","year":"2011","journal-title":"Muscle Nerve"},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"1175","DOI":"10.1007\/s12192-019-01039-2","article-title":"Coenzyme Q10 supplementation acts as antioxidant on dystrophic muscle cells","volume":"24","author":"Mizobuti","year":"2019","journal-title":"Cell Stress Chaperones"},{"key":"ref_86","doi-asserted-by":"crossref","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":"ref_87","doi-asserted-by":"crossref","first-page":"2003","DOI":"10.1113\/jphysiol.2007.148338","article-title":"N-Acetylcysteine ameliorates skeletal muscle pathophysiology in mdx mice","volume":"586","author":"Whitehead","year":"2008","journal-title":"J. Physiol."},{"key":"ref_88","doi-asserted-by":"crossref","first-page":"7093","DOI":"10.1113\/JP274229","article-title":"Pre-clinical evaluation of N-acetylcysteine reveals side effects in the mdx mouse model of Duchenne muscular dystrophy","volume":"595","author":"Pinniger","year":"2017","journal-title":"J. Physiol."},{"key":"ref_89","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1016\/j.nmd.2010.06.016","article-title":"Debio-025 is more effective than prednisone in reducing muscular pathology in mdx mice","volume":"20","author":"Wissing","year":"2010","journal-title":"Neuromuscul. Disord."},{"key":"ref_90","doi-asserted-by":"crossref","unstructured":"Brescia, M., Chao, Y.C., Koschinski, A., Tomek, J., and Zaccolo, M. (2020). Multi-Compartment, Early Disruption of cGMP and cAMP Signalling in Cardiac Myocytes from the mdx Model of Duchenne Muscular Dystrophy. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21197056"},{"key":"ref_91","doi-asserted-by":"crossref","unstructured":"Percival, J.M., Adamo, C.M., Beavo, J.A., and Froehner, S.C. (2011). Evaluation of the Therapeutic Utility of Phosphodiesterase 5A Inhibition in the mdx Mouse Model of Duchenne Muscular Dystrophy. Phosphodiesterases as Drug Targets, Springer.","DOI":"10.1007\/978-3-642-17969-3_14"},{"key":"ref_92","doi-asserted-by":"crossref","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":"ref_93","doi-asserted-by":"crossref","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":"ref_94","doi-asserted-by":"crossref","unstructured":"Hafner, P., Bonati, U., Erne, B., Schmid, M., Rubino, D., Pohlman, U., Peters, T., Rutz, E., Frank, S., and Neuhaus, C. (2016). Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study. PLoS ONE, 11.","DOI":"10.1371\/journal.pone.0147634"},{"key":"ref_95","doi-asserted-by":"crossref","first-page":"e1914171","DOI":"10.1001\/jamanetworkopen.2019.14171","article-title":"Effect of Combination L-Citrulline and Metformin Treatment on Motor Function in Patients with Duchenne Muscular Dystrophy","volume":"2","author":"Hafner","year":"2019","journal-title":"JAMA Netw. Open."},{"key":"ref_96","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":"Duboc","year":"2007","journal-title":"Am. Heart J."},{"key":"ref_97","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1016\/j.mayocp.2015.10.019","article-title":"Angiotensin-Converting Enzyme Inhibitors or Angiotensin Receptor Blockers in Patients without Heart Failure? Insights from 254,301 Patients from Randomized Trials","volume":"91","author":"Bangalore","year":"2016","journal-title":"Mayo Clin. Proc."},{"key":"ref_98","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":"Raman","year":"2015","journal-title":"Lancet Neurol."},{"key":"ref_99","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1016\/j.phrs.2013.03.003","article-title":"GLPG0492, a novel selective androgen receptor modulator, improves muscle performance in the exercised-mdx mouse model of muscular dystrophy","volume":"72","author":"Cozzoli","year":"2013","journal-title":"Pharmacol. Res."},{"key":"ref_100","doi-asserted-by":"crossref","unstructured":"Ran, N., Gao, X., Dong, X., Li, J., Lin, C., Geng, M., and Yin, H. (2020). Effects of exosome-mediated delivery of myostatin propeptide on functional recovery of mdx mice. Biomaterials, 236.","DOI":"10.1016\/j.biomaterials.2020.119826"},{"key":"ref_101","doi-asserted-by":"crossref","first-page":"4318","DOI":"10.1073\/pnas.0709144105","article-title":"Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors","volume":"105","author":"Haidet","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_102","doi-asserted-by":"crossref","first-page":"E134","DOI":"10.1002\/mus.25644","article-title":"Combined XIL-6R and urocortin-2 treatment restores MDX diaphragm muscle force","volume":"56","author":"Manning","year":"2017","journal-title":"Muscle Nerve"},{"key":"ref_103","doi-asserted-by":"crossref","first-page":"12946","DOI":"10.1096\/fj.201902102RR","article-title":"cAMP-dependent protein kinase inhibits FoxO activity and regulates skeletal muscle plasticity in mice","volume":"34","author":"Silveira","year":"2020","journal-title":"FASEB J."},{"key":"ref_104","doi-asserted-by":"crossref","first-page":"5175","DOI":"10.1113\/JP276954","article-title":"Recovery of respiratory function in mdx mice co-treated with neutralizing interleukin-6 receptor antibodies and urocortin-2","volume":"596","author":"Burns","year":"2018","journal-title":"J. Physiol."}],"container-title":["Neurology International"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2035-8377\/16\/4\/55\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T15:10:45Z","timestamp":1760109045000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2035-8377\/16\/4\/55"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,5]]},"references-count":104,"journal-issue":{"issue":"4","published-online":{"date-parts":[[2024,8]]}},"alternative-id":["neurolint16040055"],"URL":"https:\/\/doi.org\/10.3390\/neurolint16040055","relation":{},"ISSN":["2035-8377"],"issn-type":[{"value":"2035-8377","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,5]]}}}