{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,6]],"date-time":"2026-07-06T19:26:20Z","timestamp":1783365980977,"version":"3.54.6"},"reference-count":43,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T00:00:00Z","timestamp":1713225600000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T00:00:00Z","timestamp":1713225600000},"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":[[2024,4,30]]},"abstract":"<jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>\n                      Stride Velocity 95\n                      <jats:sup>th<\/jats:sup>\n                      Centile (SV95C) is the first wearable device-derived clinical outcome assessment (COA) to receive European Medicines Agency (EMA) qualification as a primary endpoint in ambulant patients with Duchenne muscular dystrophy (DMD) aged \u22654 years.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To compare SV95C\u2014in its first-ever clinical trial application as a secondary endpoint\u2014with established motor function COAs used in the trial (Four-Stair Climb [4SC] velocity, North Star Ambulatory Assessment [NSAA], and Six-Minute Walk Distance [6MWD]).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>\n                      SV95C was a secondary endpoint in a subset (\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a47) of participants in the SPITFIRE\/WN40227 trial of taldefgrobep alfa, which was discontinued due to lack of clinical benefit. Participants in the \u226448-week SV95C sub-study were 6\u201311 years old and received corticosteroids for \u22656 months pre-treatment. Pearson correlations were used to compare SV95C with the other COAs. Responsiveness and changes over time were respectively assessed via standardized response means (SRMs) based on absolute changes and mixed models for repeated measures.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>\n                      SV95C change at Week 24 was \u20130.07\u200am\/s, with limited variability (standard deviation: 0.16,\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a27). The SRM for SV95C indicated moderate responsiveness to clinical change at the earliest timepoint (Week 12,\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a46), while those of the other COAs did not indicate moderate responsiveness until Week 36 (6MWD,\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a33) or Week 48 (4SC velocity,\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a20; NSAA total score,\n                      <jats:italic toggle=\"yes\">n<\/jats:italic>\n                      \u200a=\u200a20). Baseline correlations between SV95C and other COAs were strong (\n                      <jats:italic toggle=\"yes\">r<\/jats:italic>\n                      \u200a=\u200a0.611\u20130.695). Correlations between SV95C change from baseline to Week 48 and changes in other COAs were moderate to strong (\n                      <jats:italic toggle=\"yes\">r<\/jats:italic>\n                      \u200a=\u200a0.443\u20130.678).\u2225\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Overall, SV95C demonstrated sensitivity to ambulatory decline over short intervals, low variability, and correlation with established COAs. Although the negative trial precluded demonstration of SV95C\u2019s sensitivity to drug effect, these findings support the continued use of SV95C in DMD clinical trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-230188","type":"journal-article","created":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T12:18:04Z","timestamp":1713269884000},"page":"701-714","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":18,"title":["Stride Velocity 95\n                    <sup>th<\/sup>\n                    Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"11","author":[{"given":"Michael","family":"Rabbia","sequence":"first","affiliation":[{"name":"Genentech Inc., 1 DNA Way, South San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maitea","family":"Guridi Ormazabal","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hannah","family":"Staunton","sequence":"additional","affiliation":[{"name":"Roche Products Ltd, Hexagon Place, Shire Park, 6 Falcon Way, Welwyn Garden City, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klaas","family":"Veenstra","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Damien","family":"Eggenspieler","sequence":"additional","affiliation":[{"name":"SYSNAV, 72 Rue Emile Loubet, Vernon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00e9lanie","family":"Annoussamy","sequence":"additional","affiliation":[{"name":"SYSNAV, 72 Rue Emile Loubet, Vernon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre","place":["UK"]},{"name":"University of Oxford","place":["UK"]},{"name":"University Hospital Li\u00e8ge and University of Li\u00e8ge","place":["Belgium"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Strijbos","sequence":"additional","affiliation":[{"name":"F. 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