{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T02:56:44Z","timestamp":1784689004331,"version":"3.55.0"},"reference-count":31,"publisher":"SAGE Publications","issue":"1","license":[{"start":{"date-parts":[[2024,12,8]],"date-time":"2024-12-08T00:00:00Z","timestamp":1733616000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2024,12,8]],"date-time":"2024-12-08T00:00:00Z","timestamp":1733616000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:p>\n                    Duchenne muscular dystrophy is a severe neuromuscular disorder characterized by progressive muscle degeneration resulting from mutations in the\n                    <jats:italic toggle=\"yes\">dystrophin<\/jats:italic>\n                    gene. Digital outcome measures offer a promising alternative to traditional outcome measures used in clinical trials. This review explores the development and application of digital outcome measures in Duchenne muscular dystrophy, emphasizing the feasibility, reliability, sensitivity, and validity of these measures. The stride velocity 95th centile has been validated as a robust endpoint and has been approved for use in clinical evaluation of drugs for the treatment of Duchenne muscular dystrophy by the European Medicines Agency. Although digital outcome measures have the potential to enhance the efficiency and accuracy of clinical trials, challenges such as limited sample sizes and patient compliance persist. The integration of artificial intelligence into the data analysis is in progress, but further validation is required before these analysis strategies can be incorporated into future clinical trial methodologies.\n                  <\/jats:p>","DOI":"10.1177\/22143602241296280","type":"journal-article","created":{"date-parts":[[2024,12,8]],"date-time":"2024-12-08T16:44:06Z","timestamp":1733676246000},"page":"77-85","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Digital outcome measures in Duchenne muscular dystrophy: Lessons learnt from clinical trials"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-3070-1192","authenticated-orcid":false,"given":"Camila","family":"Gonz\u00e1lez Barral","sequence":"first","affiliation":[{"name":"Sysnav, Vernon, France"},{"name":"Neuromuscular Reference Center, Department of Pediatrics, University Hospital Li\u00e8ge &amp; University of Li\u00e8ge, Li\u00e8ge, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"Neuromuscular Reference Center, Department of Pediatrics, University Hospital Li\u00e8ge &amp; University of Li\u00e8ge, Li\u00e8ge, Belgium"},{"name":"MDUK Oxford Neuromuscular Centre &amp; NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,12,8]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphys.2023.1183101"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-11796-x"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2022.916714"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2010.07.002"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2020.02.007"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1111\/ped.12348"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26763"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200478"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.1007\/s43441-021-00278-9"},{"key":"e_1_3_3_12_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26759"},{"key":"e_1_3_3_13_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27119"},{"key":"e_1_3_3_14_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25743"},{"key":"e_1_3_3_15_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.clnu.2021.06.008"},{"key":"e_1_3_3_16_2","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811413581"},{"key":"e_1_3_3_17_2","doi-asserted-by":"publisher","DOI":"10.3390\/s24041155"},{"key":"e_1_3_3_18_2","doi-asserted-by":"publisher","DOI":"10.3390\/s24041123"},{"key":"e_1_3_3_19_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2010.09.017"},{"key":"e_1_3_3_20_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0156696"},{"key":"e_1_3_3_21_2","doi-asserted-by":"publisher","DOI":"10.1063\/1.5126116"},{"key":"e_1_3_3_22_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-022-02045-1"},{"key":"e_1_3_3_23_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27335"},{"key":"e_1_3_3_24_2","doi-asserted-by":"publisher","DOI":"10.1109\/CVPR.2017.143"},{"key":"e_1_3_3_25_2","doi-asserted-by":"publisher","DOI":"10.1159\/000517411"},{"key":"e_1_3_3_26_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210743"},{"key":"e_1_3_3_27_2","unstructured":"European Medicines Agency. Final Qualification Opinion for Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy studies\u2019. 2023. [Online] Available: https:\/\/www.ema.europa.eu\/en\/documents\/scientific-guideline\/qualification-opinion-stride-velocity-95th-centile-primary-endpoint-studies-ambulatory-duchenne-muscular-dystrophy-studies_en.pdf"},{"key":"e_1_3_3_28_2","unstructured":"\u2018Sarepta Therapeutics Announces Topline Results from EMBARK a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy | Sarepta Therapeutics Inc.\u2019 Accessed: Jun. 28 2024. [Online]. Available: https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-topline-results-embark-global-0."},{"key":"e_1_3_3_29_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230188"},{"key":"e_1_3_3_30_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-023-02459-5"},{"key":"e_1_3_3_31_2","unstructured":"Dyne Therapeutics Inc. \u2018Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints\u2019. 2024. [Online] Available: https:\/\/investors.dyne-tx.com\/news-releases\/news-release-details\/dyne-therapeutics-announces-new-clinical-data-achieve-trial-dyne\/."},{"key":"e_1_3_3_32_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01956-5"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602241296280","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/22143602241296280","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602241296280","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T06:10:34Z","timestamp":1777961434000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/22143602241296280"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12,8]]},"references-count":31,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["10.1177\/22143602241296280"],"URL":"https:\/\/doi.org\/10.1177\/22143602241296280","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,12,8]]}}}