{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,22]],"date-time":"2026-08-22T04:27:20Z","timestamp":1787372840362,"version":"build-2736575974"},"reference-count":73,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T00:00:00Z","timestamp":1787184000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Wyeth Pharmaceuticals"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Muscles"],"abstract":"<jats:p>Myostatin (GDF8) is a member of the transforming growth factor-\u03b2 (TGF-\u03b2) superfamily. Myostatin gene mutations or inhibition of the Myostatin\/Activin pathway results in increased skeletal muscle mass, demonstrating its role as a negative regulator of skeletal muscle. Myostatin blockade is a promising strategy for increasing muscle mass in myopathies such as Duchenne Muscular Dystrophy (DMD); however, it faces considerable challenges in clinical translation, in part due to the progressive nature of the disease. Here we tested the ability of JA16 monoclonal antibody-mediated myostatin blockade to improve the dystrophic phenotype in newborn mdx mice (an animal model of DMD). Myostatin inhibition led to significant increases in muscle size, fiber number and cross-sectional area along with increased absolute force alongside reduced post-eccentric contraction force drop and reduced serum creatine kinase. We used the Multiparametric Muscle Improvement Score (MMIS) to objectively quantitate benefits in this preclinical study and determined that the magnitude of improvements exceeded those reported using the exact same intervention in older mdx mice treated for the same duration. This study demonstrates an age-dependent aspect of this intervention and suggests that earlier interventions may provide greater therapeutic benefits.<\/jats:p>","DOI":"10.3390\/muscles5030058","type":"journal-article","created":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T08:10:20Z","timestamp":1787213420000},"page":"58","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Age-Dependent Effect of Myostatin Blockade in the mdx Mouse Model of Duchenne Muscular Dystrophy (DMD)"],"prefix":"10.3390","volume":"5","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-1338-0415","authenticated-orcid":false,"given":"Sasha","family":"Bogdanovich","sequence":"first","affiliation":[{"name":"Department of Physiology, Pennsylvania Muscle Institute, Perelman School of Medicine, University of Pennsylvania, 415 Curie Boulevard, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emidio E.","family":"Pistilli","sequence":"additional","affiliation":[{"name":"Department of Physiology, Pennsylvania Muscle Institute, Perelman School of Medicine, University of Pennsylvania, 415 Curie Boulevard, Philadelphia, PA 19104, USA"},{"name":"School of Medicine, West Virginia University, Morgantown, WV 26506, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tejvir S.","family":"Khurana","sequence":"additional","affiliation":[{"name":"Department of Physiology, Pennsylvania Muscle Institute, Perelman School of Medicine, University of Pennsylvania, 415 Curie Boulevard, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,8,20]]},"reference":[{"key":"ref_1","unstructured":"Engel, A.G., and Franzini-Armstrong, C. (1994). Myology, McGraw-Hill. [2nd ed.]."},{"key":"ref_2","doi-asserted-by":"crossref","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":"ref_3","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/0092-8674(88)90383-2","article-title":"The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein","volume":"53","author":"Koenig","year":"1988","journal-title":"Cell"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"16717","DOI":"10.1016\/S0021-9258(17)44816-2","article-title":"Identification of a chromosome 6-encoded dystrophin-related protein","volume":"265","author":"Khurana","year":"1990","journal-title":"J. Biol. Chem."},{"key":"ref_5","doi-asserted-by":"crossref","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":"ref_6","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1038\/360591a0","article-title":"Primary structure of dystrophin-related protein","volume":"360","author":"Tinsley","year":"1992","journal-title":"Nature"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"223","DOI":"10.1038\/ng0696-223","article-title":"Characterization of DRP2, a novel human dystrophin homologue","volume":"13","author":"Roberts","year":"1996","journal-title":"Nat. Genet."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1093\/hmg\/3.5.841-a","article-title":"(CA) repeat polymorphism in the chromosome 18 encoded dystrophin-like protein","volume":"3","author":"Khurana","year":"1994","journal-title":"Hum. Mol. Genet."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"7802","DOI":"10.1074\/jbc.271.13.7802","article-title":"Isoform diversity of dystrobrevin, the murine 87-kDa postsynaptic protein","volume":"271","author":"Blake","year":"1996","journal-title":"J. Biol. Chem."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1093\/hmg\/5.4.489","article-title":"Cloning and characterization of the human homologue of a dystrophin related phosphoprotein found at the Torpedo electric organ post-synaptic membrane","volume":"5","author":"Khurana","year":"1996","journal-title":"Hum. Mol. Genet."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1016\/0092-8674(95)90344-5","article-title":"Three muscular dystrophies: Loss of cytoskeleton-extracellular matrix linkage","volume":"80","author":"Campbell","year":"1996","journal-title":"Cell"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"9161","DOI":"10.1016\/S0021-9258(18)31565-5","article-title":"Purification of dystrophin from skeletal muscle","volume":"266","author":"Ervasti","year":"1991","journal-title":"J. Biol. Chem."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"588","DOI":"10.1038\/360588a0","article-title":"Association of dystrophin-related protein with dystrophin-associated proteins in mdx mouse muscle","volume":"360","author":"Matsumura","year":"1992","journal-title":"Nature"},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1016\/0166-2236(88)90006-9","article-title":"Molecular Biology of Duchenne muscular dystrophy","volume":"11","author":"Brown","year":"1988","journal-title":"Trends Neurosci."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1146\/annurev.med.48.1.457","article-title":"Dystrophin-associated proteins and the muscular dystrophies","volume":"48","author":"Brown","year":"1997","journal-title":"Annu. Rev. Med."},{"key":"ref_16","unstructured":"Brown, S.C., and Lucy, J.A. (1997). Dystrophin: Gene, Protein and Cell Biology, Cambridge University Press."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1038\/356435a0","article-title":"Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation","volume":"356","author":"Gussoni","year":"1992","journal-title":"Nature"},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1002\/mus.880150504","article-title":"Human myoblast transplantation: Preliminary results of 4 cases","volume":"15","author":"Huard","year":"1992","journal-title":"Muscle Nerve"},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1056\/NEJM199509283331303","article-title":"Myoblast Transfer in the Treatment of Duchenne\u2019s Muscular Dystrophy","volume":"333","author":"Mendell","year":"1995","journal-title":"N. Engl. J. Med."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"1817","DOI":"10.1002\/stem.1668","article-title":"Ex vivo gene editing of the dystrophin gene in muscle stem cells mediated by peptide nucleic acid single stranded oligodeoxynucleotides induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy","volume":"32","author":"Bertoni","year":"2014","journal-title":"Stem Cells"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","article-title":"Prevention of muscular dystrophy in mice by CRISPR\/Cas9-mediated editing of germline DNA","volume":"345","author":"Long","year":"2014","journal-title":"Science"},{"key":"ref_22","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_23","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1126\/science.aad5725","article-title":"Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy","volume":"351","author":"Long","year":"2016","journal-title":"Science"},{"key":"ref_24","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_25","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.stem.2016.01.021","article-title":"A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells","volume":"18","author":"Young","year":"2016","journal-title":"Cell Stem Cell"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"e124297","DOI":"10.1172\/jci.insight.124297","article-title":"AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice","volume":"3","author":"Hakim","year":"2018","journal-title":"JCI Insight"},{"key":"ref_28","doi-asserted-by":"crossref","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":"ref_29","doi-asserted-by":"crossref","first-page":"1626","DOI":"10.1161\/01.CIR.0000089371.11664.27","article-title":"Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart","volume":"108","author":"Yue","year":"2003","journal-title":"Circulation"},{"key":"ref_30","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_31","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_32","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1089\/hum.2019.255","article-title":"Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy","volume":"32","author":"Potter","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"ref_33","doi-asserted-by":"crossref","unstructured":"Manini, A., Abati, E., Nuredini, A., Corti, S., and Comi, G.P. (2021). 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_34","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1089\/hum.2015.107","article-title":"Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy","volume":"26","author":"Nance","year":"2015","journal-title":"Hum. Gene Ther."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1097\/WCO.0000000000000241","article-title":"Viral vector-mediated gene therapies","volume":"28","author":"Hollinger","year":"2015","journal-title":"Curr. Opin. Neurol."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"213","DOI":"10.3233\/JND-180351","article-title":"Eteplirsen Investigators and the CINRG DNHS Investigators. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy","volume":"6","author":"Khan","year":"2019","journal-title":"J. Neuromuscul. Dis."},{"key":"ref_37","doi-asserted-by":"crossref","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":"ref_38","doi-asserted-by":"crossref","first-page":"2123","DOI":"10.1212\/WNL.0000000000003217","article-title":"Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy","volume":"87","author":"Griggs","year":"2016","journal-title":"Neurology"},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"1473","DOI":"10.1038\/s41591-019-0604-2","article-title":"Surrogate gene therapy for muscular dystrophy","volume":"25","author":"Davies","year":"2019","journal-title":"Nat. Med."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"e2304","DOI":"10.1212\/WNL.0000000000012910","article-title":"Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis","volume":"97","author":"Broomfield","year":"2021","journal-title":"Neurology"},{"key":"ref_41","doi-asserted-by":"crossref","unstructured":"Sienkiewicz, D., Kulak, W., Okurowska-Zawada, B., Paszko-Patej, G., Wojtkowski, J., Sochon, K., Kalinowska, A., Okulczyk, K., Sienkiewicz, J., and McEachern, E. (2017). Efficacy and the Safety of Granulocyte Colony-Stimulating Factor Treatment in Patients with Muscular Dystrophy: A Non-Randomized Clinical Trial. Front. Neurol., 8.","DOI":"10.3389\/fneur.2017.00566"},{"key":"ref_42","doi-asserted-by":"crossref","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":"ref_43","doi-asserted-by":"crossref","first-page":"9306","DOI":"10.1073\/pnas.151270098","article-title":"Regulation of myostatin activity and muscle growth","volume":"98","author":"Lee","year":"2001","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"40235","DOI":"10.1074\/jbc.M004356200","article-title":"Myostatin, a negative regulator of muscle growth, functions by inhibiting myoblast proliferation","volume":"275","author":"Thomas","year":"2000","journal-title":"J. Biol. Chem."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"251","DOI":"10.3109\/08977190109029114","article-title":"GDF-8 propeptide binds to GDF-8 and antagonizes biological activity by inhibiting GDF-8 receptor binding","volume":"18","author":"Tries","year":"2001","journal-title":"Growth Factors"},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"561","DOI":"10.1006\/bbrc.2000.4159","article-title":"Myostatin regulates cell survival during C2C12 myogenesis","volume":"280","author":"Rios","year":"2001","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1146\/annurev-physiol-012422-112116","article-title":"Myostatin: A Skeletal Muscle Chalone","volume":"85","author":"Lee","year":"2023","journal-title":"Annu. Rev. Physiol."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1038\/nrd1085","article-title":"Pharmacological strategies for muscular dystrophy","volume":"2","author":"Khurana","year":"2003","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"1535","DOI":"10.1007\/s11010-024-05120-y","article-title":"Therapeutic applications and challenges in myostatin inhibition for enhanced skeletal muscle mass and functions","volume":"480","author":"Wetzlich","year":"2025","journal-title":"Mol. Cell. Biochem."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"1265","DOI":"10.1038\/ijo.2009.162","article-title":"The effects of a soluble activin type IIB receptor on obesity and insulin sensitivity","volume":"33","author":"Akpan","year":"2009","journal-title":"Int. J. Obes."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"R96","DOI":"10.1152\/ajpregu.00138.2009","article-title":"Pretreatment with a soluble activin type IIB receptor\/Fc fusion protein improves hypoxia-induced muscle dysfunction","volume":"298","author":"Pistilli","year":"2010","journal-title":"Am. J. Physiol.-Regul. Integr. Comp. Physiol."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1016\/j.ajpath.2010.11.071","article-title":"Targeting the activin type IIB receptor to improve muscle mass and function in the mdx mouse model of Duchenne muscular dystrophy","volume":"178","author":"Pistilli","year":"2011","journal-title":"Am. J. Pathol."},{"key":"ref_53","doi-asserted-by":"crossref","unstructured":"Cadena, S.M., Bogdanovich, S., Khurana, T.S., Pullen, A., Pearsall, R.S., Curran, E., Faucette, R., Lane, J., Seehra, J., and Lachey, J.L. (2026). ACE-031, a soluble activin type IIB receptor, increases muscle mass and strength in the common marmoset (Callithrix jacchus). PLoS ONE, 21.","DOI":"10.1371\/journal.pone.0342666"},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1097\/WCO.0000000000000853","article-title":"The elusive promise of myostatin inhibition for muscular dystrophy","volume":"33","author":"Wagner","year":"2020","journal-title":"Curr. Opin. Neurol."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"418","DOI":"10.1038\/nature01154","article-title":"Functional improvement of dystrophic muscle by myostatin blockade","volume":"420","author":"Bogdanovich","year":"2002","journal-title":"Nature"},{"key":"ref_56","doi-asserted-by":"crossref","unstructured":"Bogdanovich, S., and Pistilli, E.E. (2012). From Basic Research to Clinical Trials: Preclinical Trial Evaluation in Mouse Models, Muscular Dystrophy, InTechOpen.","DOI":"10.5772\/30920"},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1007\/s40120-023-00570-w","article-title":"The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy","volume":"13","author":"Muntoni","year":"2024","journal-title":"Neurol. Ther."},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"e2033457","DOI":"10.1001\/jamanetworkopen.2020.33457","article-title":"Effect of Bimagrumab vs Placebo on Body Fat Mass Among Adults with Type 2 Diabetes and Obesity: A Phase 2 Randomized Clinical Trial","volume":"4","author":"Heymsfield","year":"2021","journal-title":"JAMA Netw. Open"},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1038\/s41591-026-04204-0","article-title":"Bimagrumab plus semaglutide alone or in combination for the treatment of obesity: A randomized phase 2 trial","volume":"32","author":"Heymsfield","year":"2026","journal-title":"Nat. Med."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"49831","DOI":"10.1074\/jbc.M204291200","article-title":"Myostatin inhibits myoblast differentiation by down-regulating MyoD expression","volume":"277","author":"Langley","year":"2002","journal-title":"J. Biol. Chem."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"1135","DOI":"10.1083\/jcb.200207056","article-title":"Myostatin negatively regulates satellite cell activation and self-renewal","volume":"162","author":"McCroskery","year":"2003","journal-title":"J. Cell Biol."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"2151","DOI":"10.1113\/jphysiol.2011.226001","article-title":"Myostatin inhibition induces muscle fibre hypertrophy prior to satellite cell activation","volume":"590","author":"Wang","year":"2012","journal-title":"J. Physiol."},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"E3231","DOI":"10.1073\/pnas.1204017109","article-title":"Cripto regulates skeletal muscle regeneration and modulates satellite cell determination by antagonizing myostatin","volume":"109","author":"Guardiola","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"431","DOI":"10.1055\/s-2007-972660","article-title":"The effect of training status on the serum creatine kinase response, soreness and muscle function following resistance exercise","volume":"18","author":"Vincent","year":"1997","journal-title":"Int. J. Sports Med."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.nbd.2008.03.008","article-title":"Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy","volume":"31","author":"Grounds","year":"2008","journal-title":"Neurobiol. Dis."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1002\/mus.21211","article-title":"Preclinical drug trials in the mdx mouse: Assessment of reliable and sensitive outcome measures","volume":"39","author":"Spurney","year":"2009","journal-title":"Muscle Nerve"},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"502","DOI":"10.1016\/j.nmd.2009.05.003","article-title":"Developing standard procedures for murine and canine efficacy studies of DMD therapeutics: Report of two expert workshops on \u201cPre-clinical testing for Duchenne dystrophy\u201d: Washington DC, October 27th\u201328th 2007 and Zurich, June 30th\u2013July 1st 2008","volume":"19","author":"Nagaraju","year":"2009","journal-title":"Neuromuscul. Disord."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1007\/s003359900551","article-title":"Behavioral and functional analysis of mouse phenotype: SHIRPA, a proposed protocol for comprehensive phenotype assessment","volume":"8","author":"Rogers","year":"1997","journal-title":"Mamm. Genome"},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1016\/j.nmd.2009.06.368","article-title":"Reliability of the North Star Ambulatory Assessment in a multicentric setting","volume":"19","author":"Mazzone","year":"2009","journal-title":"Neuromuscul. Disord."},{"key":"ref_70","doi-asserted-by":"crossref","unstructured":"Muntoni, F., Guglieri, M., Mah, J.K., Wagner, K.R., Brandsema, J.F., Butterfield, R.J., McDonald, C.M., Mayhew, A.G., Palmer, J.P., and Marraffino, S. (2022). Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial. PLoS ONE, 17.","DOI":"10.1371\/journal.pone.0272858"},{"key":"ref_71","doi-asserted-by":"crossref","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":"ref_72","doi-asserted-by":"crossref","first-page":"3333","DOI":"10.1093\/hmg\/11.26.3333","article-title":"Prevention of pathology in mdx mice by expression of utrophin: Analysis using an inducible transgenic expression system","volume":"11","author":"Squire","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"13856","DOI":"10.1073\/pnas.0405972101","article-title":"Heregulin ameliorates the dystrophic phenotype in mdx mice","volume":"101","author":"Krag","year":"2004","journal-title":"Proc. Natl. Acad. Sci. USA"}],"container-title":["Muscles"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2813-0413\/5\/3\/58\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,22]],"date-time":"2026-08-22T04:15:58Z","timestamp":1787372158000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2813-0413\/5\/3\/58"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,20]]},"references-count":73,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2026,9]]}},"alternative-id":["muscles5030058"],"URL":"https:\/\/doi.org\/10.3390\/muscles5030058","relation":{},"ISSN":["2813-0413"],"issn-type":[{"value":"2813-0413","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,8,20]]}}}