{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T02:20:20Z","timestamp":1773195620730,"version":"3.50.1"},"reference-count":72,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,3,27]],"date-time":"2024-03-27T00:00:00Z","timestamp":1711497600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["CPT Pharmacom &amp; Syst Pharma"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Duchenne muscular dystrophy (DMD) is a rare X\u2010linked recessive disorder characterized by loss\u2010of\u2010function mutations in the gene encoding dystrophin. These mutations lead to progressive functional deterioration including muscle weakness, respiratory insufficiency, and musculoskeletal deformities. Three\u2010dimensional gait analysis (3DGA) has been used as a tool to analyze gait pathology through the quantification of altered joint kinematics, kinetics, and muscle activity patterns. Among 3DGA indices, the Gait Profile Score (GPS), has been used as a sensitive overall measure to detect clinically relevant changes in gait patterns in children with DMD. To enhance our understanding of the clinical translation of 3DGA, we report here the development of a population nonlinear mixed\u2010effect model that jointly describes the disease progression of the 3DGA index, GPS, and the functional endpoint, North Star Ambulatory Assessment (NSAA). The final model consists of a quadratic structure for GPS progression and a linear structure for GPS\u2010NSAA correlation. Our model was able to capture the improvement in function in GPS and NSAA in younger subjects, as well as the decline of function in older subjects. Furthermore, the model predicted NSAA (CFB) at 1\u2009year reasonably well for DMD subjects \u22647\u2009years old at baseline. The model tended to slightly underpredict the decline in NSAA after 1\u2009year for those &gt;7\u2009years old at baseline, but the prediction summary statistics were well maintained within the standard deviation of observed data. Quantitative models such as this may help answer clinically relevant questions to facilitate the development of novel therapies in DMD.<\/jats:p>","DOI":"10.1002\/psp4.13126","type":"journal-article","created":{"date-parts":[[2024,3,28]],"date-time":"2024-03-28T00:10:12Z","timestamp":1711584612000},"page":"891-903","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Population longitudinal analysis of Gait Profile Score and North Star Ambulatory Assessment in children with Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-0995-0659","authenticated-orcid":false,"given":"Jiexin","family":"Deng","sequence":"first","affiliation":[{"name":"School of Nursing and Health Henan University  Kaifeng China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7249-4965","authenticated-orcid":false,"given":"Fangli","family":"Liu","sequence":"additional","affiliation":[{"name":"School of Nursing and Health Henan University  Kaifeng China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhifen","family":"Feng","sequence":"additional","affiliation":[{"name":"School of Nursing and Health Henan University  Kaifeng China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhigang","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Orthopedics First Affiliated Hospital of Henan University  Kaifeng China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,3,27]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.2174\/138161210790883471"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10928-013-9318-0"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30025-5"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30026-7"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(02)07815-7"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.200307-885ST"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1155\/2013\/406163"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1183\/09031936.06.00059906"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2018-0333I"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamacardio.2016.4910"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev.med.58.011706.144703"},{"issue":"5","key":"e_1_2_11_14_1","article-title":"Corticosteroids for the treatment of Duchenne muscular dystrophy","volume":"2016","author":"Matthews E","year":"2016","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2004.05.001"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.880170405"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2006.01.010"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2012.12.008"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01837-x"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.1097\/PHM.0b013e3181e72207"},{"key":"e_1_2_11_22_1","first-page":"9","article-title":"Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy","volume":"31","author":"Angelini C","year":"2012","journal-title":"Acta Myol"},{"key":"e_1_2_11_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2014.11.011"},{"key":"e_1_2_11_24_1","unstructured":"EMA.EMA confirms recommendation for non\u2010renewal of authorisation of Duchenne muscular dystrophy medicine Translarna. European Medicines Agency.2024.https:\/\/www.ema.europa.eu\/en\/news\/ema\u2010confirms\u2010recommendation\u2010non\u2010renewal\u2010authorisation\u2010duchenne\u2010muscular\u2010dystrophy\u2010medicine\u2010translarna"},{"key":"e_1_2_11_25_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-016-0657-1"},{"key":"e_1_2_11_26_1","unstructured":"FDA.FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation. FDA News Release.2019.https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010grants\u2010accelerated\u2010approval\u2010first\u2010targeted\u2010treatment\u2010rare\u2010duchenne\u2010muscular\u2010dystrophy\u2010mutation"},{"key":"e_1_2_11_27_1","unstructured":"FDA.FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation. FDA News Release.2020.https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010approves\u2010targeted\u2010treatment\u2010rare\u2010duchenne\u2010muscular\u2010dystrophy\u2010mutation"},{"key":"e_1_2_11_28_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-021-01512-2"},{"key":"e_1_2_11_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_11_30_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21544"},{"key":"e_1_2_11_31_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.06.368"},{"key":"e_1_2_11_32_1","doi-asserted-by":"publisher","DOI":"10.1002\/pri.520"},{"key":"e_1_2_11_33_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23807"},{"key":"e_1_2_11_34_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23808"},{"key":"e_1_2_11_35_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2012-303902"},{"key":"e_1_2_11_36_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318225ab2e"},{"key":"e_1_2_11_37_1","doi-asserted-by":"publisher","DOI":"10.1371\/currents.RRN1297"},{"key":"e_1_2_11_38_1","first-page":"149","article-title":"The NorthStar ambulatory assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials","volume":"87","author":"Ricotti V","year":"2016","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"e_1_2_11_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.10.007"},{"key":"e_1_2_11_40_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2020.05.031"},{"key":"e_1_2_11_41_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2008.06.002"},{"key":"e_1_2_11_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2016.05.013"},{"key":"e_1_2_11_43_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2010.01.010"},{"key":"e_1_2_11_44_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2015.04.019"},{"key":"e_1_2_11_45_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2009.05.020"},{"key":"e_1_2_11_46_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.earlhumdev.2023.105843"},{"key":"e_1_2_11_47_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2018.03.020"},{"key":"e_1_2_11_48_1","doi-asserted-by":"publisher","DOI":"10.2340\/16501977-2507"},{"key":"e_1_2_11_49_1","doi-asserted-by":"publisher","DOI":"10.1080\/09638288.2018.1506946"},{"key":"e_1_2_11_50_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gaitpost.2022.12.007"},{"key":"e_1_2_11_51_1","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1016\/j.nmd.2013.06.577","article-title":"Three\u2010dimensional gait analysis of Duchenne muscular dystrophy: a trial to evaluate the therapeutic effect of RNA\/ENA chimera antisense oligonucleotide that induces dystrophin exon 45 skipping, 18th International Congress of the World Muscle Society","volume":"23","author":"Y T","year":"2013","journal-title":"Neuromuscul Disord"},{"key":"e_1_2_11_52_1","doi-asserted-by":"publisher","DOI":"10.3390\/s24041123"},{"key":"e_1_2_11_53_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnhum.2022.861136"},{"key":"e_1_2_11_54_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0108205"},{"key":"e_1_2_11_55_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0253882"},{"key":"e_1_2_11_56_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01758-9"},{"key":"e_1_2_11_57_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20812"},{"key":"e_1_2_11_58_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.07.011"},{"key":"e_1_2_11_59_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210682"},{"key":"e_1_2_11_60_1","first-page":"43","article-title":"Read\u2010through approach for stop mutations in Duchenne muscular dystrophy. An update","volume":"40","author":"Politano L","year":"2021","journal-title":"Acta Myol"},{"key":"e_1_2_11_61_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.1003222"},{"key":"e_1_2_11_62_1","doi-asserted-by":"publisher","DOI":"10.11005\/jbm.2020.27.3.151"},{"key":"e_1_2_11_63_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2022.950651"},{"key":"e_1_2_11_64_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10928-020-09671-7"},{"key":"e_1_2_11_65_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12753"},{"key":"e_1_2_11_66_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12921"},{"key":"e_1_2_11_67_1","volume-title":"Gait in Rehabilitation","author":"Smidt G","year":"1990"},{"key":"e_1_2_11_68_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0162463"},{"key":"e_1_2_11_69_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2018.00849"},{"key":"e_1_2_11_70_1","doi-asserted-by":"publisher","DOI":"10.1097\/MRR.0000000000000304"},{"key":"e_1_2_11_71_1","unstructured":"EMA.Qualification opinion for stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne muscular dystrophy studies. European Medicines Agency.2023.https:\/\/www.ema.europa.eu\/en\/documents\/scientific\u2010guideline\/qualification\u2010opinion\u2010stride\u2010velocity\u201095th\u2010centile\u2010primary\u2010endpoint\u2010studies\u2010ambulatory\u2010duchenne\u2010muscular\u2010dystrophy\u2010studies_en.pdf"},{"key":"e_1_2_11_72_1","first-page":"668","article-title":"Modeling disease progression trajectories from longitudinal observational data","volume":"2020","author":"Kwon BC","year":"2020","journal-title":"AMIA Annu Symp Proc"},{"key":"e_1_2_11_73_1","doi-asserted-by":"crossref","unstructured":"CouronneR VidailhetM CorvolJC LehericyS DurrlemanS.Learning disease progression models with longitudinal data and missing values. 2019 IEEE 16th International Symposium on Biomedical Imaging (ISBI 2019) Venice Italy 2019 1033\u201310372019.","DOI":"10.1109\/ISBI.2019.8759198"}],"container-title":["CPT: Pharmacometrics &amp; Systems Pharmacology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/psp4.13126","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,7,19]],"date-time":"2025-07-19T16:43:55Z","timestamp":1752943435000},"score":1,"resource":{"primary":{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/10.1002\/psp4.13126"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3,27]]},"references-count":72,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2024,5]]}},"alternative-id":["10.1002\/psp4.13126"],"URL":"https:\/\/doi.org\/10.1002\/psp4.13126","archive":["Portico"],"relation":{},"ISSN":["2163-8306","2163-8306"],"issn-type":[{"value":"2163-8306","type":"print"},{"value":"2163-8306","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,3,27]]},"assertion":[{"value":"2023-10-28","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-01","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-27","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}