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Träfflista för sökning "WFRF:(Masson E) "

Search: WFRF:(Masson E)

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  • 2017
  • swepub:Mat__t
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  • Niemi, MEK, et al. (author)
  • 2021
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  • Mercuri, E., et al. (author)
  • Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
  • 2020
  • In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
  • Journal article (peer-reviewed)abstract
    • 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 (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-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: 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). Results & conclusion: Kaplan-Meier 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 <= 0.05). There were no DMD genotype-phenotype/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. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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  • Result 1-10 of 112
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journal article (105)
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peer-reviewed (105)
other academic/artistic (5)
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Brown, A. (37)
Colijn, A. P. (36)
Aprile, E. (36)
Budnik, R. (36)
Landsman, H. (36)
Lang, R. F. (36)
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Masbou, J. (36)
Murra, M. (36)
Dos Santos, J. M. F. (36)
Schumann, M. (36)
Selvi, M. (36)
Zhang, Y. (36)
Diglio, S. (35)
Agostini, F. (35)
Arneodo, F. (35)
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Ni, K. (35)
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Plante, G. (35)
Rupp, N. (35)
Sartorelli, G. (35)
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Thers, D. (35)
Wei, Y. (35)
Weinheimer, C. (35)
Gao, F. (35)
Masson, D. (35)
Pienaar, J. (35)
Manfredini, A. (34)
Conrad, Jan (34)
Bruenner, S. (34)
Alfonsi, M. (34)
Cardoso, J. M. R. (34)
Cussonneau, J. P. (34)
Lindner, M. (34)
Messina, M. (34)
Schreiner, J. (34)
Simgen, H. (34)
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