Sökning: onr:"swepub:oai:gup.ub.gu.se/287625" > Ataluren use in pat...
Fältnamn | Indikatorer | Metadata |
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000 | 02929naa a2200445 4500 | |
001 | oai:gup.ub.gu.se/287625 | |
003 | SwePub | |
008 | 240910s2019 | |||||||||||000 ||eng| | |
024 | 7 | a https://gup.ub.gu.se/publication/2876252 URI |
024 | 7 | a https://doi.org/10.2217/cer-2019-00862 DOI |
040 | a (SwePub)gu | |
041 | a eng | |
042 | 9 SwePub | |
072 | 7 | a ref2 swepub-contenttype |
072 | 7 | a art2 swepub-publicationtype |
100 | 1 | a Muntoni, F.4 aut |
245 | 1 0 | a Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry |
264 | 1 | b Becaris Publishing Limited,c 2019 |
520 | a Aim: 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 (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population. Patients & methods: Patients will be followed up from enrollment for >= 5 years or until study withdrawal. Results & conclusion: As of 9 July 2018, 213 DMD boys were enrolled from 11 countries. Mean (standard deviation) ages at first symptoms and at study treatment start were 2.7 (1.7) years 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. | |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicin0 (SwePub)3022 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicine0 (SwePub)3022 hsv//eng |
700 | 1 | a Desguerre, I.4 aut |
700 | 1 | a Guglieri, M.4 aut |
700 | 1 | a Osorio, A. N.4 aut |
700 | 1 | a Kirschner, J.4 aut |
700 | 1 | a Tulinius, Mar,d 1953u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xtulim |
700 | 1 | a Buccella, F.4 aut |
700 | 1 | a Elfring, G.4 aut |
700 | 1 | a Werner, C.4 aut |
700 | 1 | a Schilling, T.4 aut |
700 | 1 | a Trifillis, P.4 aut |
700 | 1 | a Zhang, O.4 aut |
700 | 1 | a Delage, A.4 aut |
700 | 1 | a Santos, C. L.4 aut |
700 | 1 | a Mercuri, E.4 aut |
710 | 2 | a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org |
773 | 0 | t Journal of Comparative Effectiveness Researchd : Becaris Publishing Limitedg 8:14, s. 1187-1200q 8:14<1187-1200x 2042-6305x 2042-6313 |
856 | 4 | u https://doi.org/10.2217/cer-2019-0086 |
856 | 4 8 | u https://gup.ub.gu.se/publication/287625 |
856 | 4 8 | u https://doi.org/10.2217/cer-2019-0086 |
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