{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T04:15:16Z","timestamp":1791951316733,"version":"4.3.4"},"reference-count":32,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2022,2,14]],"date-time":"2022-02-14T00:00:00Z","timestamp":1644796800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2022,8]]},"abstract":"<jats:sec><jats:title>Aim<\/jats:title><jats:p>To correlate the North Star Ambulatory Assessment (NSAA) and timed rise from floor (TRF) recorded at age of expected peak with age at loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD).<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>Male children with DMD enrolled in the UK North Start Network database were included according to the following criteria: follow\u2010up longer than 3 years, one NSAA record between 6 years and 7 years 6 months (baseline), at least one visit when older than 8 years. Data about corticosteroid treatment, LOA, genotype, NSAA, and TRF were analysed. Age at LOA among the different groups based on NSAA and TRF was determined by log\u2010rank tests. Cox proportional hazard models were used for multivariable analysis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 293 patients from 13 different centres were included. Mean (SD) age at first and last visit was 5 years 6 months (1 year 2 months) and 12 years 8 months (2 years 11 months) (median follow\u2010up 7 years 4 months). Higher NSAA and lower TRF at baseline were associated with older age at LOA (<jats:italic>p<\/jats:italic>&lt;0.001). Patients scoring NSAA 32 to 34 had a probability of 0.61 of being ambulant when older than 13 years compared with 0.34 for those scoring 26 to 31. In multivariable analysis, NSAA, TRF, and corticosteroid daily regimen (vs intermittent) were all independently associated with outcome (<jats:italic>p<\/jats:italic>=0.01).<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Higher functional abilities at peak are associated with older age at LOA in DMD. This information is important for counselling families. These baseline measures should also be considered when designing clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.15176","type":"journal-article","created":{"date-parts":[[2022,2,14]],"date-time":"2022-02-14T08:43:45Z","timestamp":1644828225000},"page":"979-988","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":46,"title":["Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy"],"prefix":"10.1111","volume":"64","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6539-5360","authenticated-orcid":false,"given":"Alberto A.","family":"Zambon","sequence":"first","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"},{"name":"Neuromuscular Repair Unit Institute of Experimental Neurology (InSpe) Division of Neuroscience IRCCS Ospedale San Raffaele  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vandana","family":"Ayyar Gupta","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deborah","family":"Ridout","sequence":"additional","affiliation":[{"name":"Population, Policy and Practice Research and Teaching Department UCL Great Ormond Street Institute of Child Health  London UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adnan\u00a0Y.","family":"Manzur","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanni","family":"Baranello","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Trucco","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"},{"name":"Children\u2019s Sleep Medicine Evelina Children Hospital \u2010 Paediatric Respiratory Department Royal Brompton Hospital Guy\u2019s and St Thomas\u2019 Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health &amp; Great Ormond Street Hospital  London UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"The UK Northstar Clinical Network","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,2,14]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90039-U"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01430-8"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0160195"},{"key":"e_1_2_9_6_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_9_7_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0221097"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2012-303902"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318225ab2e"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0151445"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0108205"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0218683"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23902"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23808"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(15)60025-3"},{"key":"e_1_2_9_18_1","first-page":"1475","article-title":"Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options","volume":"11","author":"LoMauro A","year":"2015","journal-title":"Ther Clin Risk Manag"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000010170"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.05.016"},{"key":"e_1_2_9_21_1","article-title":"On behalf of the North Star clinical n. Duchenne muscular dystrophy patients lacking the dystrophin isoforms Dp140 and Dp71 and mouse models lacking Dp140 have a more severe motor phenotype.","author":"Chesshyre M","year":"2021","journal-title":"medRxiv"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1484"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2020.08.013"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2011.03939.x"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/pri.520"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0232870"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2020.08.063"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0052512"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0164684"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI91445"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001950"},{"key":"e_1_2_9_33_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-150100"}],"container-title":["Developmental Medicine &amp; Child Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/dmcn.15176","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/dmcn.15176","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/dmcn.15176","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,18]],"date-time":"2023-08-18T09:55:01Z","timestamp":1692352501000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/dmcn.15176"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,2,14]]},"references-count":32,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2022,8]]}},"alternative-id":["10.1111\/dmcn.15176"],"URL":"https:\/\/doi.org\/10.1111\/dmcn.15176","archive":["Portico"],"relation":{},"ISSN":["0012-1622","1469-8749"],"issn-type":[{"value":"0012-1622","type":"print"},{"value":"1469-8749","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,2,14]]},"assertion":[{"value":"2021-10-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-01-17","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-02-14","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}