{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T05:23:30Z","timestamp":1790832210676,"version":"4.1.0"},"reference-count":51,"publisher":"Becaris Publishing Limited","issue":"5","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2020,4]]},"abstract":"<jats:p>\n                    <jats:bold>Aim:<\/jats:bold>\n                    Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype\u2013phenotype\/\u2013ataluren benefit correlations and ataluren safety.\n                    <jats:bold>Patients &amp; methods:<\/jats:bold>\n                    Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018).\n                    <jats:bold>Results &amp; conclusion:<\/jats:bold>\n                    Kaplan\u2013Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p \u2264 0.05). There were no DMD genotype\u2013phenotype\/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice.\n                    <jats:bold>ClinicalTrials.gov identifier:<\/jats:bold>\n                    NCT02369731.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:bold>ClinicalTrials.gov identifier:<\/jats:bold>\n                    NCT02369731.\n                  <\/jats:p>","DOI":"10.2217\/cer-2019-0171","type":"journal-article","created":{"date-parts":[[2020,1,30]],"date-time":"2020-01-30T06:51:36Z","timestamp":1580367096000},"page":"341-360","source":"Crossref","is-referenced-by-count":110,"title":["Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study"],"prefix":"10.57264","volume":"9","author":[{"given":"Eugenio","family":"Mercuri","sequence":"first","affiliation":[{"name":"Department of Pediatric Neurology, Catholic University, Rome, Italy"},{"name":"Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre &amp; MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health &amp; Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9s Nascimento","family":"Osorio","sequence":"additional","affiliation":[{"name":"Hospital Sant Joan de D\u00e9u Unidad de Patolog\u00eda Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00e1r","family":"Tulinius","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Gothenburg University, Queen Silvia Children\u2019s Hospital, Gothenburg, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Filippo","family":"Buccella","sequence":"additional","affiliation":[{"name":"Parent Project Italy APS, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren P","family":"Morgenroth","sequence":"additional","affiliation":[{"name":"Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gordish-Dressman","sequence":"additional","affiliation":[{"name":"Center for Genetic Medicine, Children\u2019s National Health System &amp; the George Washington, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Jiang","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Panayiota","family":"Trifillis","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Zhu","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allan","family":"Kristensen","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio L","family":"Santos","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik K","family":"Henricson","sequence":"additional","affiliation":[{"name":"University of California Davis School of Medicine, Davis, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis School of Medicine, Davis, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Desguerre","sequence":"additional","affiliation":[{"name":"APHP Necker \u2013 Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the STRIDE","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"CINRG Duchenne Natural History Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"3","key":"e_1_3_8_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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