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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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- Fritsch, Daniel J., et al.
(författare)
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Experimental and Computational Study of 2D Smooth Wall Turbulent Boundary Layers in Pressure Gradient
- 2022
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Ingår i: AIAA Science and Technology Forum and Exposition, AIAA SciTech Forum 2022. - Reston, Virginia : American Institute of Aeronautics and Astronautics.
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Konferensbidrag (refereegranskat)abstract
- This paper describes a collaborative experimental and computational study of smooth wall boundary layers in a systematic family of favorable and adverse pressure gradients. The objective is to advance turbulence modeling of these flows, in particular the effects of pressure gradients that can be classified as non-equilibrium. This collaboration is a component of the larger NATO AVT-349 Research Task Group. Experiments under this effort are conducted at Virginia Tech and computational efforts are presented from Virginia Tech, the German Aerospace Center (DLR), the University of Melbourne, Chalmers University of Technology, the Maritime Research Institute Netherlands (MARIN) in conjunction with the University of Lisbon Instituto Superior Técnico (IST) (MARIN/IST), and the Sirehna Naval Group. This paper describes some of the key elements of the experimental and computational approaches, the efforts made for cross-discipline collaboration, verification, and validation, and reports on some initial results and findings. The agreement between various RANS solutions and RANS turbulence models and between RANS solutions and experiment are generally good, but questions remain as to the efficacy of RANS modeling for non-equilibrium boundary layer flows and some potential directions for future investigations are suggested.
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