{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T07:48:58Z","timestamp":1793346538716,"version":"4.5.0"},"reference-count":34,"publisher":"Becaris Publishing Limited","issue":"10","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2025,10]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            To report the efficacy and safety of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the phase III, 72-week, placebo-controlled period of Study 041.\n            <jats:bold>Materials &amp; methods:<\/jats:bold>\n            Inclusion criteria: boys with nmDMD aged \u22655\u00a0years, on a stable corticosteroid regimen for \u226512\u00a0months, and baseline 6-minute walk distance (6MWD) \u2265150\u00a0m. Randomization: 1:1, ataluren (40\u00a0mg\/kg\/day):placebo. Primary end point: slope of 6MWD change (average rate of change). Secondary end points: changes in 6MWD, time to 10% persistent worsening in 6MWD, North Star Ambulatory Assessment score, timed function tests and safety. Study populations: intention-to-treat; patients aged \u22657 to \u226416\u00a0years with baseline 6MWD \u2265300\u00a0m and stand from supine \u22655\u00a0s; patients with baseline 6MWD 300\u2013400\u00a0m.\n            <jats:bold>Results:<\/jats:bold>\n            In the intention-to-treat population (n\u00a0=\u00a0359), over 72\u00a0weeks, ataluren reduced the rate of 6MWD decline by 21% (p = 0.0248), reduced the average 6MWD change (p = 0.0248), delayed time to 10% persistent worsening in 6MWD (p = 0.0078), and reduced North Star Ambulatory Assessment total score decline (p = 0.0235), change in 10\u00a0m walk\/run time (p\u00a0=\u00a00.0422) and change in time to climb four stairs (p = 0.0293) versus placebo. In the 6MWD 300\u2013400\u00a0m subgroup (n\u00a0=\u00a0169), ataluren reduced the rate of 6MWD decline by 30% (p = 0.0310) versus placebo. Ataluren treatment benefits were seen in secondary end points in this subgroup, except for change in time to descend four stairs. In the 6MWD \u2265300\u00a0m and time to stand from supine \u22655s subgroup (n\u00a0=\u00a0185), there was a 9% slower rate of 6MWD decline for ataluren versus placebo over 72\u00a0weeks (p = 0.3626). Ataluren reduced change in time to climb four stairs (p = 0.0179) versus placebo in this subgroup; no treatment benefits were seen for other secondary end points. Ataluren was well tolerated (serious adverse events: ataluren, 7.1%; placebo, 6.8%); no deaths occurred.\n            <jats:bold>Conclusion:<\/jats:bold>\n            Long-term ataluren treatment has a favorable benefit\u2013risk profile, slowing motor function decline in the largest phase III nmDMD study to date.\n          <\/jats:p>","DOI":"10.57264\/cer-2024-0238","type":"journal-article","created":{"date-parts":[[2025,8,22]],"date-time":"2025-08-22T08:54:28Z","timestamp":1755852868000},"source":"Crossref","is-referenced-by-count":3,"title":["Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial"],"prefix":"10.57264","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2635-2752","authenticated-orcid":false,"given":"Dmitry","family":"Vlodavets","sequence":"first","affiliation":[{"name":"Russian Children Neuromuscular Center, Veltischev Clinical Pediatric &amp; 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Departments of Pediatrics &amp; Laboratory Medicine, &amp; Translational Research Center of Neuromuscular Diseases, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hirofumi","family":"Komaki","sequence":"additional","affiliation":[{"name":"Translational Medical Center, National Center of Neurology &amp; Psychiatry, Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Statland","sequence":"additional","affiliation":[{"name":"University of Kansas Medical Center, Kansas City, KS, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra Prufer","family":"de Queiroz Campos Araujo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juliana","family":"Gurgel-Giannetti","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Faculty of Medicine, Federal University of Minas Gerais, Belo Horizonte, Minas Gerais, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasuhiro","family":"Takeshima","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Hyogo Medical University, Nishinomiya, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rosa E","family":"Escobar Cedillo","sequence":"additional","affiliation":[{"name":"Instituto Nacional de Rehabilitacion, Mexico City, Mexico"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Karachunski","sequence":"additional","affiliation":[{"name":"University of Minnesota, Minneapolis, MN, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazuhiro","family":"Haginoya","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, Miyagi Children\u2019s Hospital, Sendai, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vinay","family":"Penematsa","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., Warren, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Connie","family":"Chou","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., Warren, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula","family":"Williams","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., Warren, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Werner","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Germany GmbH, Frankfurt, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis School of Medicine, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"3","key":"e_1_3_9_3_2","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management","volume":"17","author":"Birnkrant DJ","year":"2018","unstructured":"Birnkrant DJ, Bushby K, Bann CM Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. 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