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Ataluren use in pat...
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
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Muntoni, F. (author)
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Desguerre, I. (author)
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Guglieri, M. (author)
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Osorio, A. N. (author)
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Kirschner, J. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Buccella, F. (author)
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Elfring, G. (author)
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Werner, C. (author)
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Schilling, T. (author)
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Trifillis, P. (author)
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Zhang, O. (author)
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Delage, A. (author)
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Santos, C. L. (author)
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Mercuri, E. (author)
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(creator_code:org_t)
- Becaris Publishing Limited, 2019
- 2019
- English.
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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 8:14, s. 1187-1200
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Abstract
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine (hsv//eng)
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- ref (subject category)
- art (subject category)
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- By the author/editor
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Muntoni, F.
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Desguerre, I.
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Guglieri, M.
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Osorio, A. N.
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Kirschner, J.
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Tulinius, Mar, 1 ...
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show more...
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Buccella, F.
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Elfring, G.
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Werner, C.
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Schilling, T.
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Trifillis, P.
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Zhang, O.
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Delage, A.
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Santos, C. L.
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Mercuri, E.
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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Journal of Compa ...
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University of Gothenburg