{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,31]],"date-time":"2026-07-31T22:48:52Z","timestamp":1785538132059,"version":"3.56.0"},"reference-count":25,"publisher":"Becaris Publishing Limited","issue":"14","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2019,10]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            Strategic Targeting of Registries and International Database of Excellence (STRIDE)\u00a0is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population.\n            <jats:bold>Patients &amp; methods:<\/jats:bold>\n            Patients will be followed up from enrollment for \u22655\u00a0years or until study withdrawal.\n            <jats:bold>Results &amp; conclusion:<\/jats:bold>\n            As of 9 July 2018, 213 DMD boys were enrolled from 11\u00a0countries. Mean\u00a0(standard deviation) ages at first symptoms and at study treatment start were 2.7 (1.7)\u00a0years and 9.8 (3.7) years, respectively. Corticosteroids were used by 190 patients (89.2%) before data cut-off. Mean (standard deviation) ataluren exposure was 639.0 (362.9) days. Six patients withdrew. STRIDE is the first drug registry for patients with DMD and represents the largest real-world registry of patients with nmDMD to date.\n          <\/jats:p>","DOI":"10.2217\/cer-2019-0086","type":"journal-article","created":{"date-parts":[[2019,8,15]],"date-time":"2019-08-15T11:21:31Z","timestamp":1565868091000},"page":"1187-1200","source":"Crossref","is-referenced-by-count":34,"title":["Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry"],"prefix":"10.57264","volume":"8","author":[{"given":"Francesco","family":"Muntoni","sequence":"first","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":"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"}]},{"given":"Michela","family":"Guglieri","sequence":"additional","affiliation":[{"name":"The John Walton Muscular Dystrophy Research Centre, Newcastle University &amp; Newcastle Upon Tyne Hospitals, Newcastle Upon Tyne, 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":"Janbernd","family":"Kirschner","sequence":"additional","affiliation":[{"name":"Medical Center \u2013 University of Freiburg, Faculty of Medicine, Freiburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00e1r","family":"Tulinius","sequence":"additional","affiliation":[{"name":"Department of\u00a0Pediatrics, 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":"Gary","family":"Elfring","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, 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":"Traci","family":"Schilling","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":"Olivia","family":"Zhang","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abdallah","family":"Delage","sequence":"additional","affiliation":[{"name":"PTC Therapeutics Switzerland GmbH, Zug, Switzerland"}],"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":"Eugenio","family":"Mercuri","sequence":"additional","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"}]}],"member":"36897","reference":[{"issue":"8","key":"e_1_3_8_3_2","doi-asserted-by":"crossref","first-page":"682","DOI":"10.1016\/j.nmd.2013.05.008","article-title":"195th ENMC International Workshop: newborn screening for Duchenne muscular dystrophy December 14\u201316, 2012, Naarden, The Netherlands","volume":"23","author":"Ellis JA","year":"2013","unstructured":"Ellis JA , Vroom E , Muntoni F . 195th ENMC International Workshop: newborn screening for Duchenne muscular dystrophy December 14\u201316, 2012, Naarden, The Netherlands. Neuromuscul. Disord. 23(8), 682\u2013689 (2013).","journal-title":"Neuromuscul. Disord."},{"issue":"1","key":"e_1_3_8_4_2","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/S1474-4422(09)70271-6","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management","volume":"9","author":"Bushby K","year":"2010","unstructured":"Bushby K , Finkel R , Birnkrant DJ Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 9(1), 77\u201393 (2010).","journal-title":"Lancet Neurol."},{"key":"e_1_3_8_5_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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