{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,18]],"date-time":"2025-11-18T09:27:14Z","timestamp":1763458034289,"version":"3.40.5"},"reference-count":14,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2021,9,2]],"date-time":"2021-09-02T00:00:00Z","timestamp":1630540800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013223","name":"PTC Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013223","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2021,11]]},"abstract":"Abstract<\/jats:title>Introduction\/Aims<\/jats:title>In comparative studies, treatment effects are typically evaluated at a specific time point. When data are collected periodically, an alternative, clinically meaningful approach could be used to assess the totality of treatment effects. We applied a well\u2010developed analytical procedure for evaluating longitudinal treatment effects using North Star Ambulatory Assessment (NSAA) data for illustration.<\/jats:p><\/jats:sec>Methods<\/jats:title>The NSAA comprises 17 scorable items\/outcomes that measure changes in motor function. Using NSAA data from the published ataluren phase 3, randomized, placebo\u2010controlled trial (NCT01826487), cumulative counts of failures to perform each item (transition from 2\/1 [able\/impaired] to 0 [unable]) were collected at specified time points for each patient over 48\u2009wk. Treatment group\u2010wise mean cumulative item failure count curves were constructed, comparing ataluren versus placebo and deflazacort versus prednisone\/prednisolone among placebo\u2010treated patients. The steeper the curve, the worse the outcome. A clinically meaningful summary of the between\u2010group difference was provided for each comparison.<\/jats:p><\/jats:sec>Results<\/jats:title>The curve was uniformly steeper for placebo than ataluren after 16\u2009wk and for prednisone\/prednisolone than deflazacort after 8\u00a0wk. The two curves in each comparison continued to diverge thereafter, indicating sustained treatment benefits over time. Using a unique analytical approach, cumulative failure rates were reduced, on average, by 27% for ataluren versus placebo (rate ratio, 0.73; 95% confidence interval [CI], 0.55\u20130.97; p<\/jats:italic>\u00a0=\u2009.027) and 28% for deflazacort versus prednisone\/prednisolone (rate ratio, 0.72; 95% CI, 0.53\u20130.96; p<\/jats:italic>\u00a0=\u2009.028).<\/jats:p><\/jats:sec>Discussion<\/jats:title>Unlike fixed\u2010time analyses, this analytical approach enabled demonstration of cumulative, longitudinal treatment effects over time using repeatedly measured NSAA observations.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27396","type":"journal-article","created":{"date-parts":[[2021,8,12]],"date-time":"2021-08-12T10:00:20Z","timestamp":1628762420000},"page":"614-619","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment<\/scp>"],"prefix":"10.1002","volume":"64","author":[{"given":"Craig M.","family":"McDonald","sequence":"first","affiliation":[{"name":"Physical Medicine & Rehabilitation University of California Davis Davis California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lee\u2010Jen","family":"Wei","sequence":"additional","affiliation":[{"name":"Harvard University Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6440-3376","authenticated-orcid":false,"given":"Kevin M.","family":"Flanigan","sequence":"additional","affiliation":[{"name":"Nationwide Children's Hospital Columbus Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gary","family":"Elfring","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc South Plainfield New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Panayiota","family":"Trifillis","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc South Plainfield New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"University College London Great Ormond Street Institute of Child Health and NIHR Biomedical Research Centre London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"the ACT DMD Clinical Evaluator Training Group","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"the ACT DMD Study Group","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,9,2]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1136\/jmedgenet-2015-103387"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(87)90579-4"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.59"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-med-120611-144851"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_2_10_8_1","unstructured":"European Medicines Agency.Translarna\u2122 summary of product characteristics. Accessed February 11 2021www.ema.europa.eu\/documents\/product-information\/translarna-epar-product-information_en.pdf"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)31611-2"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.06.368"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/pri.520"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/1467-9868.00259"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/ejhf.1139"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1715971"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26191"}],"container-title":["Muscle & Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27396","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27396","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27396","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,25]],"date-time":"2023-08-25T14:41:03Z","timestamp":1692974463000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27396"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,9,2]]},"references-count":14,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2021,11]]}},"alternative-id":["10.1002\/mus.27396"],"URL":"https:\/\/doi.org\/10.1002\/mus.27396","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"type":"print","value":"0148-639X"},{"type":"electronic","value":"1097-4598"}],"subject":[],"published":{"date-parts":[[2021,9,2]]},"assertion":[{"value":"2020-11-25","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-08-07","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-09-02","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}