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- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)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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- Jungebluth, P, et al.
(författare)
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Tracheal tissue engineering in rats
- 2014
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Ingår i: Nature protocols. - : Springer Science and Business Media LLC. - 1750-2799 .- 1754-2189. ; 9:9, s. 2164-2179
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Tidskriftsartikel (refereegranskat)
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| 11. |
- Lemon, G, et al.
(författare)
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The development of the bioartificial lung
- 2014
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Ingår i: British medical bulletin. - : Oxford University Press (OUP). - 1471-8391 .- 0007-1420. ; 110:1, s. 35-45
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Tidskriftsartikel (refereegranskat)
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- Sjoqvist, S, et al.
(författare)
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Publisher Correction: Experimental orthotopic transplantation of a tissue-engineered oesophagus in rats
- 2018
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Ingår i: Nature communications. - : Springer Science and Business Media LLC. - 2041-1723. ; 9, s. 16208-
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Tidskriftsartikel (refereegranskat)abstract
- Nature Communications 5: Article number: 3562 (2014); Published online: 15 April 2014; Updated: 10 April 2018 The original HTML version of this Article had an incorrect article number of 4562; it should have been 3562. This has now been corrected in the HTML; the PDF version of the Article was correct from the time of publication.
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| 21. |
- Sjoqvist, S, et al.
(författare)
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Retraction: Experimental orthotopic transplantation of a tissue-engineered oesophagus in rats
- 2017
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Ingår i: Nature communications. - : Springer Science and Business Media LLC. - 2041-1723. ; 8, s. 15077-
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Tidskriftsartikel (refereegranskat)abstract
- Nature Communications 5: Article number: 3562 (2014); Published 15 April 2014; Updated 21 March 2017 This Article is retracted by the authors. Nature Communications previously issued an Editorial Expression of Concern (http://www.nature.com/articles/ncomms13310) related to this Article, following the publication of a report commissioned by The Karolinska Institute and prepared by the Expert Group for Misconduct in Research at the Swedish Central Ethical Review Board.
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