{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T02:36:12Z","timestamp":1793414172336,"version":"4.5.0"},"reference-count":38,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,1,4]],"date-time":"2024-01-04T00:00:00Z","timestamp":1704326400000},"content-version":"vor","delay-in-days":3,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100014943","name":"sarepta therapeutics inc","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Nat Med"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Limb-girdle muscular dystrophy 2E\/R4 is caused by mutations in the \u03b2-sarcoglycan (\n                    <jats:italic>SGCB<\/jats:italic>\n                    ) gene, leading to SGCB deficiency and consequent muscle loss. We developed a gene therapy approach based on functional replacement of the deficient SCB protein. Here we report interim results from a first-in-human, open-label, nonrandomized, phase\u20091\/2 trial evaluating the safety and efficacy of bidridistrogene xeboparvovec, an adeno-associated virus-based gene therapy containing a codon-optimized, full-length human\n                    <jats:italic>SGCB<\/jats:italic>\n                    transgene. Patients aged 4\u201315\u2009years with confirmed\n                    <jats:italic>SGCB<\/jats:italic>\n                    mutations at both alleles received one intravenous infusion of either 1.85\u2009\u00d7\u200910\n                    <jats:sup>13<\/jats:sup>\n                    vector genome copies\u2009kg\n                    <jats:sup>\u2212<\/jats:sup>\n                    <jats:sup>1<\/jats:sup>\n                    (Cohort\u20091,\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20093) or 7.41\u2009\u00d7\u200910\n                    <jats:sup>13<\/jats:sup>\n                    vector gene copies\u2009kg\n                    <jats:sup>\u22121<\/jats:sup>\n                    (Cohort\u20092,\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20093). Primary endpoint was safety, and secondary endpoint was change in SGCB expression in skeletal muscle from baseline to Day\u200960. We report interim Year\u20092 results (trial ongoing). The most frequent treatment-related adverse events were vomiting (four of six\u2009patients) and gamma-glutamyl transferase increase (three of six\u2009patients). Serious adverse events resolved with standard therapies. Robust SGCB expression was observed: Day\u200960 mean (s.d.) percentage of normal expression 36.2% (2.7%) in Cohort\u20091 and 62.1% (8.7%) in Cohort\u20092. Post hoc exploratory analysis showed preliminary motor improvements using the North Star Assessment for Limb-girdle Type Muscular Dystrophies maintained through Year\u20092. The 2-year safety and efficacy of bidridistrogene xeboparvovec support clinical development advancement. Further studies are necessary to confirm the long-term safety and efficacy of this gene therapy. ClinicalTrials.gov registration:\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT03652259\">NCT03652259<\/jats:ext-link>\n                    .\n                  <\/jats:p>","DOI":"10.1038\/s41591-023-02730-9","type":"journal-article","created":{"date-parts":[[2024,1,4]],"date-time":"2024-01-04T06:02:08Z","timestamp":1704348128000},"page":"199-206","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":32,"title":["Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E\/R4: phase 1\/2 trial results"],"prefix":"10.1038","volume":"30","author":[{"given":"Jerry R.","family":"Mendell","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric R.","family":"Pozsgai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Lewis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danielle A.","family":"Griffin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4206-0557","authenticated-orcid":false,"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1236-4293","authenticated-orcid":false,"given":"Kelly J.","family":"Lehman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen","family":"Church","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0573-9504","authenticated-orcid":false,"given":"Natalie F.","family":"Reash","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2800-3855","authenticated-orcid":false,"given":"Megan A.","family":"Iammarino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenna","family":"Sabo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachael","family":"Potter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Neuhaus","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoxi","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Herb","family":"Stevenson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8966-5177","authenticated-orcid":false,"given":"Louise R.","family":"Rodino-Klapac","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,1,4]]},"reference":[{"key":"2730_CR1","doi-asserted-by":"publisher","first-page":"S7","DOI":"10.3233\/JND-150105","volume":"2","author":"AP Murphy","year":"2015","unstructured":"Murphy, A. P. & Straub, V. The classification, natural history and treatment of the limb girdle muscular dystrophies. J. Neuromuscul. Dis. 2, S7\u2013S19 (2015).","journal-title":"J. Neuromuscul. Dis."},{"key":"2730_CR2","first-page":"1599","volume":"25","author":"MP Wicklund","year":"2019","unstructured":"Wicklund, M. P. The limb-girdle muscular dystrophies. Continuum (Minneap. Minn.) 25, 1599\u20131618 (2019).","journal-title":"Continuum (Minneap. Minn.)"},{"key":"2730_CR3","doi-asserted-by":"publisher","first-page":"294","DOI":"10.1038\/nrneurol.2016.35","volume":"12","author":"R Thompson","year":"2016","unstructured":"Thompson, R. & Straub, V. Limb-girdle muscular dystrophies \u2013 international collaborations for translational research. Nat. Rev. Neurol. 12, 294\u2013309 (2016).","journal-title":"Nat. Rev. Neurol."},{"key":"2730_CR4","doi-asserted-by":"publisher","first-page":"702","DOI":"10.1016\/j.nmd.2018.05.007","volume":"28","author":"V Straub","year":"2018","unstructured":"Straub, V., Murphy, A. & Udd, B. Limb girdle muscular dystrophies \u2013 nomenclature and reformed classification. Neuromuscul. Disord. 28, 702\u2013710 (2018).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR5","first-page":"119","volume":"33","author":"C Angelini","year":"2014","unstructured":"Angelini, C., Tasca, E., Nascimbeni, A. C. & Fanin, M. Muscle fatigue, nNOS and muscle fiber atrophy in limb girdle muscular dystrophy. Acta Myol. 33, 119\u2013126 (2014).","journal-title":"Acta Myol."},{"key":"2730_CR6","doi-asserted-by":"publisher","first-page":"768","DOI":"10.1590\/0004-282X20140126","volume":"72","author":"MA Albuquerque","year":"2014","unstructured":"Albuquerque, M. A. et al. Clinical aspects of patients with sarcoglycanopathies under steroids therapy. Arq. Neuropsiquiatr. 72, 768\u2013772 (2014).","journal-title":"Arq. Neuropsiquiatr."},{"key":"2730_CR7","doi-asserted-by":"publisher","first-page":"341","DOI":"10.1111\/cge.13597","volume":"96","author":"PB Winckler","year":"2019","unstructured":"Winckler, P. B. et al. Clinicogenetic lessons from 370 patients with autosomal recessive limb-girdle muscular dystrophy. Clin. Genet. 96, 341\u2013353 (2019).","journal-title":"Clin. Genet."},{"key":"2730_CR8","doi-asserted-by":"publisher","first-page":"e0153095","DOI":"10.1371\/journal.pone.0153095","volume":"11","author":"A Fayssoil","year":"2016","unstructured":"Fayssoil, A. et al. Natural history of cardiac and respiratory involvement, prognosis and predictive factors for long-term survival in adult patients with limb girdle muscular dystrophies type 2C and 2D. PLoS ONE 11, e0153095 (2016).","journal-title":"PLoS ONE"},{"key":"2730_CR9","doi-asserted-by":"publisher","first-page":"995","DOI":"10.1097\/01.jnen.0000235854.77716.6c","volume":"65","author":"SA Moore","year":"2006","unstructured":"Moore, S. A. et al. Limb-girdle muscular dystrophy in the United States. J. Neuropathol. Exp. Neurol. 65, 995\u20131003 (2006).","journal-title":"J. Neuropathol. Exp. Neurol."},{"key":"2730_CR10","doi-asserted-by":"publisher","first-page":"e28","DOI":"10.1017\/S1462399409001203","volume":"11","author":"D Sandon\u00e0","year":"2009","unstructured":"Sandon\u00e0, D. & Betto, R. Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects. Expert Rev. Mol. Med. 11, e28 (2009).","journal-title":"Expert Rev. Mol. Med."},{"key":"2730_CR11","doi-asserted-by":"publisher","first-page":"176","DOI":"10.1212\/WNL.52.1.176","volume":"52","author":"C Angelini","year":"1999","unstructured":"Angelini, C. et al. The clinical spectrum of sarcoglycanopathies. Neurology 52, 176\u2013179 (1999).","journal-title":"Neurology"},{"key":"2730_CR12","doi-asserted-by":"publisher","first-page":"266","DOI":"10.1038\/ng1195-266","volume":"11","author":"CG B\u00f6nnemann","year":"1995","unstructured":"B\u00f6nnemann, C. G. et al. Beta-sarcoglycan (A3b) mutations cause autosomal recessive muscular dystrophy with loss of the sarcoglycan complex. Nat. Genet. 11, 266\u2013273 (1995).","journal-title":"Nat. Genet."},{"key":"2730_CR13","doi-asserted-by":"publisher","first-page":"1772","DOI":"10.1212\/WNL.0000000000001519","volume":"84","author":"C Semplicini","year":"2015","unstructured":"Semplicini, C. et al. Clinical and genetic spectrum in limb-girdle muscular dystrophy type 2E. Neurology 84, 1772\u20131781 (2015).","journal-title":"Neurology"},{"key":"2730_CR14","doi-asserted-by":"publisher","first-page":"178","DOI":"10.1016\/S0960-8966(00)00174-7","volume":"11","author":"L Politano","year":"2001","unstructured":"Politano, L. et al. Evaluation of cardiac and respiratory involvement in sarcoglycanopathies. Neuromuscul. Disord. 11, 178\u2013185 (2001).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR15","doi-asserted-by":"publisher","first-page":"1196","DOI":"10.1001\/archneur.65.9.1196","volume":"65","author":"ML Sveen","year":"2008","unstructured":"Sveen, M. L., Thune, J. J., K\u00f8ber, L. & Vissing, J. Cardiac involvement in patients with limb-girdle muscular dystrophy type 2 and Becker muscular dystrophy. Arch. Neurol. 65, 1196\u20131201 (2008).","journal-title":"Arch. Neurol."},{"key":"2730_CR16","doi-asserted-by":"publisher","first-page":"303","DOI":"10.1016\/S0960-8966(02)00280-8","volume":"13","author":"M Fanin","year":"2003","unstructured":"Fanin, M., Melacini, P., Boito, C., Pegoraro, E. & Angelini, C. LGMD2E patients risk developing dilated cardiomyopathy. Neuromuscul. Disord. 13, 303\u2013309 (2003).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR17","doi-asserted-by":"publisher","first-page":"473","DOI":"10.1002\/(SICI)1097-4598(199904)22:4<473::AID-MUS8>3.0.CO;2-5","volume":"22","author":"P Melacini","year":"1999","unstructured":"Melacini, P. et al. Heart involvement in muscular dystrophies due to sarcoglycan gene mutations. Muscle Nerve 22, 473\u2013479 (1999).","journal-title":"Muscle Nerve"},{"key":"2730_CR18","doi-asserted-by":"publisher","first-page":"381","DOI":"10.1080\/21678707.2017.1307731","volume":"5","author":"M Carotti","year":"2017","unstructured":"Carotti, M., Fecchio, C. & Sandon\u00e0, D. Emerging therapeutic strategies for sarcoglycanopathy. Expert Opin. Orphan Drugs 5, 381\u2013396 (2017).","journal-title":"Expert Opin. Orphan Drugs"},{"key":"2730_CR19","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1080\/14712598.2020.1725469","volume":"20","author":"DR Asher","year":"2020","unstructured":"Asher, D. R. et al. Clinical development on the frontier: gene therapy for Duchenne muscular dystrophy. Expert Opin. Biol. Ther. 20, 263\u2013274 (2020).","journal-title":"Expert Opin. Biol. Ther."},{"key":"2730_CR20","doi-asserted-by":"publisher","first-page":"855","DOI":"10.1016\/j.ymthe.2017.02.013","volume":"25","author":"ER Pozsgai","year":"2017","unstructured":"Pozsgai, E. R., Griffin, D. A., Heller, K. N., Mendell, J. R. & Rodino-Klapac, L. R. Systemic AAV-mediated \u03b2-sarcoglycan delivery targeting cardiac and skeletal muscle ameliorates histological and functional deficits in LGMD2E mice. Mol. Ther. 25, 855\u2013869 (2017).","journal-title":"Mol. Ther."},{"key":"2730_CR21","doi-asserted-by":"publisher","first-page":"737","DOI":"10.1089\/hum.2016.141","volume":"28","author":"DA Zygmunt","year":"2017","unstructured":"Zygmunt, D. A., Crowe, K. E., Flanigan, K. M. & Martin, P. T. Comparison of serum rAAV serotype-specific antibodies in patients with Duchenne muscular dystrophy, Becker muscular dystrophy, inclusion body myositis, or GNE myopathy. Hum. Gene Ther. 28, 737\u2013746 (2017).","journal-title":"Hum. Gene Ther."},{"key":"2730_CR22","doi-asserted-by":"publisher","first-page":"320","DOI":"10.1038\/sj.mt.6300027","volume":"15","author":"MZ Salva","year":"2007","unstructured":"Salva, M. Z. et al. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol. Ther. 15, 320\u2013329 (2007).","journal-title":"Mol. Ther."},{"key":"2730_CR23","doi-asserted-by":"publisher","first-page":"539","DOI":"10.1586\/ehm.11.48","volume":"4","author":"D Raj","year":"2011","unstructured":"Raj, D., Davidoff, A. M. & Nathwani, A. C. Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges. Expert Rev. Hematol. 4, 539\u2013549 (2011).","journal-title":"Expert Rev. Hematol."},{"key":"2730_CR24","doi-asserted-by":"publisher","first-page":"1648","DOI":"10.1038\/mt.2008.171","volume":"16","author":"DM McCarty","year":"2008","unstructured":"McCarty, D. M. Self-complementary AAV vectors; advances and applications. Mol. Ther. 16, 1648\u20131656 (2008).","journal-title":"Mol. Ther."},{"key":"2730_CR25","doi-asserted-by":"publisher","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","volume":"377","author":"JR Mendell","year":"2017","unstructured":"Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713\u20131722 (2017).","journal-title":"N. Engl. J. Med."},{"key":"2730_CR26","doi-asserted-by":"publisher","first-page":"1122","DOI":"10.1001\/jamaneurol.2020.1484","volume":"77","author":"JR Mendell","year":"2020","unstructured":"Mendell, J. R. et al. Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol. 77, 1122\u20131131 (2020).","journal-title":"JAMA Neurol."},{"key":"2730_CR27","doi-asserted-by":"publisher","first-page":"2696","DOI":"10.1093\/brain\/awaa228","volume":"143","author":"J Alonso-P\u00e9rez","year":"2020","unstructured":"Alonso-P\u00e9rez, J. et al. New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy. Brain 143, 2696\u20132708 (2020).","journal-title":"Brain"},{"key":"2730_CR28","doi-asserted-by":"publisher","first-page":"307","DOI":"10.3233\/JND-190403","volume":"6","author":"SA Al-Zaidy","year":"2019","unstructured":"Al-Zaidy, S. A. et al. AVXS-101 (onasemnogene abeparvovec) for SMA1: comparative study with a prospective natural history cohort. J. Neuromuscul. Dis. 6, 307\u2013317 (2019).","journal-title":"J. Neuromuscul. Dis."},{"key":"2730_CR29","doi-asserted-by":"publisher","first-page":"65","DOI":"10.1080\/105294199277860","volume":"6","author":"JR Dixon Jr.","year":"1998","unstructured":"Dixon, J. R. Jr. The International Conference on Harmonization Good Clinical Practice guideline. Qual. Assur. 6, 65\u201374 (1998).","journal-title":"Qual. Assur."},{"key":"2730_CR30","doi-asserted-by":"publisher","first-page":"57","DOI":"10.1038\/gt.2015.80","volume":"23","author":"ER Pozsgai","year":"2016","unstructured":"Pozsgai, E. R., Griffin, D. A., Heller, K. N., Mendell, J. R. & Rodino-Klapac, L. R. beta-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice. Gene Ther. 23, 57\u201366 (2016).","journal-title":"Gene Ther."},{"key":"2730_CR31","doi-asserted-by":"publisher","first-page":"452","DOI":"10.1016\/j.nmd.2017.02.007","volume":"27","author":"LN Alfano","year":"2017","unstructured":"Alfano, L. N. et al. The 100-meter timed test: normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials. Neuromuscul. Disord. 27, 452\u2013457 (2017).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR32","doi-asserted-by":"publisher","first-page":"717","DOI":"10.1002\/mus.21945","volume":"43","author":"MA Tarnopolsky","year":"2011","unstructured":"Tarnopolsky, M. A., Pearce, E., Smith, K. & Lach, B. Suction-modified Bergstr\u00f6m muscle biopsy technique: experience with 13,500 procedures. Muscle Nerve 43, 717\u2013725 (2011).","journal-title":"Muscle Nerve"},{"key":"2730_CR33","doi-asserted-by":"publisher","first-page":"e2146","DOI":"10.1212\/WNL.0000000000005680","volume":"90","author":"JS Charleston","year":"2018","unstructured":"Charleston, J. S. et al. Eteplirsen treatment for Duchenne muscular dystrophy: exon skipping and dystrophin production. Neurology 90, e2146\u2013e2154 (2018).","journal-title":"Neurology"},{"key":"2730_CR34","unstructured":"Rasband, W. S. ImageJ (1997\u20132018) version 1.54F; https:\/\/imagej.nih.gov\/ij\/"},{"key":"2730_CR35","doi-asserted-by":"publisher","first-page":"967","DOI":"10.1002\/ana.26044","volume":"89","author":"MB Jacobs","year":"2021","unstructured":"Jacobs, M. B. et al. Assessing dysferlinopathy patients over three years with a new motor scale. Ann. Neurol. 89, 967\u2013978 (2021).","journal-title":"Ann. Neurol."},{"key":"2730_CR36","first-page":"S145","volume":"27","author":"M James","year":"2017","unstructured":"James, M. et al. North Star Assessment for dysferlinopathy: longitudinal performance in the clinical outcome study of dysferlinopathy. Neuromuscul. Disord. 27, S145 (2017).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR37","doi-asserted-by":"publisher","first-page":"S88","DOI":"10.1016\/j.nmd.2020.08.146","volume":"30","author":"N Miller","year":"2020","unstructured":"Miller, N. et al. Limb girdle muscular dystrophies: P.143 measuring motor function in LGMD2E: determining which functional outcomes are the most responsive to change in a 1-year study. Neuromuscul. Disord. 30, S88 (2020).","journal-title":"Neuromuscul. Disord."},{"key":"2730_CR38","doi-asserted-by":"publisher","first-page":"e461","DOI":"10.1212\/WNL.0000000000006858","volume":"92","author":"U Moore","year":"2019","unstructured":"Moore, U. et al. Assessment of disease progression in dysferlinopathy: a 1-year cohort study. Neurology 92, e461\u2013e474 (2019).","journal-title":"Neurology"}],"container-title":["Nature Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41591-023-02730-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41591-023-02730-9","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41591-023-02730-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,22]],"date-time":"2024-01-22T07:17:31Z","timestamp":1705907851000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41591-023-02730-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1]]},"references-count":38,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,1]]}},"alternative-id":["2730"],"URL":"https:\/\/doi.org\/10.1038\/s41591-023-02730-9","relation":{},"ISSN":["1078-8956","1546-170X"],"issn-type":[{"value":"1078-8956","type":"print"},{"value":"1546-170X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,1]]},"assertion":[{"value":"12 August 2022","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"20 November 2023","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 January 2024","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"J.R.M. received financial support from Sarepta Therapeutics, Inc. while at NCH at the time of the study and currently is an employee of Sarepta Therapeutics, Inc. E.R.P., S.L., D.A.G., S.N., R.P., X.L., H.S. and L.R.R.-K. are, or have been, employees of Sarepta Therapeutics, Inc. and may own stock\/options in the company. L.P.L. and L.N.A. received fees from Sarepta Therapeutics, Inc. for licensure of the LGMD natural history dataset and served on the LGMD advisory board. L.P.L., L.N.A. and N.F.R. perform outcome measures training. B.S., K.J.L., K.C., N.F.R. and M.A.I. have no competing interests to declare and J.R.M with L.R.R.-K., are coinventors of bidridistrogene xeboparvovec.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}