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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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| 4. |
- Menkveld, Albert J., et al.
(författare)
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Nonstandard Errors
- 2024
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Ingår i: JOURNAL OF FINANCE. - : Wiley-Blackwell. - 0022-1082 .- 1540-6261. ; 79:3, s. 2339-2390
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Tidskriftsartikel (refereegranskat)abstract
- In statistics, samples are drawn from a population in a data-generating process (DGP). Standard errors measure the uncertainty in estimates of population parameters. In science, evidence is generated to test hypotheses in an evidence-generating process (EGP). We claim that EGP variation across researchers adds uncertainty-nonstandard errors (NSEs). We study NSEs by letting 164 teams test the same hypotheses on the same data. NSEs turn out to be sizable, but smaller for more reproducible or higher rated research. Adding peer-review stages reduces NSEs. We further find that this type of uncertainty is underestimated by participants.
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| 5. |
- Chavanne, A. V., et al.
(författare)
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Individual-Level Prediction of Exposure Therapy Outcome Using Structural and Functional MRI Data in Spider Phobia: A Machine-Learning Study
- 2023
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Ingår i: Depression and Anxiety. - 1091-4269. ; 2023
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Tidskriftsartikel (refereegranskat)abstract
- Machine-learning prediction studies have shown potential to inform treatment stratification, but recent efforts to predict psychotherapy outcomes with clinical routine data have only resulted in moderate prediction accuracies. Neuroimaging data showed promise to predict treatment outcome, but previous prediction attempts have been exploratory and reported small clinical sample sizes. Herein, we aimed to examine the incremental predictive value of neuroimaging data in contrast to clinical and demographic data alone (for which results were previously published), using a two-level multimodal ensemble machine-learning strategy. We used pretreatment structural and task-based fMRI data to predict virtual reality exposure therapy outcome in a bicentric sample of N=190 patients with spider phobia. First, eight 1st-level random forest classifications were conducted using separate data modalities (clinical questionnaire scores and sociodemographic data, cortical thickness and gray matter volumes, functional activation, connectivity, connectivity-derived graph metrics, and BOLD signal variance). Then, the resulting predictions were used to train a 2nd-level classifier that produced a final prediction. No 1st-level or 2nd-level classifier performed above chance level except BOLD signal variance, which showed potential as a contributor to higher-level prediction from multiple regions across the brain (1st-level balanced accuracy=0.63). Overall, neuroimaging data did not provide any incremental accuracy for treatment outcome prediction in patients with spider phobia with respect to clinical and sociodemographic data alone. Thus, we advise caution in the interpretation of prediction performances from small-scale, single-site patient samples. Larger multimodal datasets are needed to further investigate individual-level neuroimaging predictors of therapy response in anxiety disorders.
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| 6. |
- Sommer, Natascha, et al.
(författare)
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Bypassing mitochondrial complex III using alternative oxidase inhibits acute pulmonary oxygen sensing
- 2020
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Ingår i: Science Advances. - : American Association for the Advancement of Science. - 2375-2548. ; 6:16
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Tidskriftsartikel (refereegranskat)abstract
- Mitochondria play an important role in sensing both acute and chronic hypoxia in the pulmonary vasculature, but their primary oxygen-sensing mechanism and contribution to stabilization of the hypoxia-inducible factor (HIF) remains elusive. Alteration of the mitochondrial electron flux and increased superoxide release from complex III has been proposed as an essential trigger for hypoxic pulmonary vasoconstriction (HPV). We used mice expressing a tunicate alternative oxidase, AOX, which maintains electron flux when respiratory complexes III and/or IV are inhibited. Respiratory restoration by AOX prevented acute HPV and hypoxic responses of pulmonary arterial smooth muscle cells (PASMC), acute hypoxia-induced redox changes of NADH and cytochrome c, and superoxide production. In contrast, AOX did not affect the development of chronic hypoxia-induced pulmonary hypertension and HIF-1α stabilization. These results indicate that distal inhibition of the mitochondrial electron transport chain in PASMC is an essential initial step for acute but not chronic oxygen sensing.
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| 7. |
- Brackmann, Christian, et al.
(författare)
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Simultaneous vibrational and pure rotational coherent anti-Stokes Raman spectroscopy for temperature and multispecies concentration measurements demonstrated in sooting flames
- 2002
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Ingår i: Applied Optics. - 2155-3165. ; 41:3, s. 564-572
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Tidskriftsartikel (refereegranskat)abstract
- The potential of measuring temperature and multiple species concentrations (N-2, O-2, CO) by use of combined vibrational coherent anti-Stokes Raman spectroscopy (CARS) and pure rotational CARS has been investigated. This was achieved with only one Nd:YAG laser and one dye laser together with a single spectrograph and CCD camera. From measurements in premixed sooting C2H4-air flames it was possible to evaluate temperatures from both vibrational CARS and rotational CARS, spectra, O-2 concentration from the rotational CARS spectra, and CO concentration from the vibrational CARS spectra. Quantitative results from premixed sooting C2H4-air flames are presented, and the uncertainties in the results as well as the possibility of extending the combined CARS technique fur probing of additional species are discussed. (C) 2002 Optical Society of America.
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| 8. |
- Buldyreva, J, et al.
(författare)
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Linewidth modelling of C2H2-N-2 mixtures tested by rotational CARS measurements
- 2006
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Ingår i: Journal of Raman Spectroscopy. - : Wiley. - 1097-4555 .- 0377-0486. ; 37:6, s. 647-654
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Tidskriftsartikel (refereegranskat)abstract
- The acetylene-nitrogen system is investigated in a combined theoretical and experimental effort in view of its thermometry applications. Accurate values of the rotational Raman linewidths of acetylene are of crucial importance for the computation of spectra from rotational coherent anti-Stokes Raman scattering (CARS) used for temperature and concentration evaluations. Since the direct measurements of these linewidths are not available, they are rigorously calculated by a semi-classical Robert-Bonamy formalism employing exact trajectories. The computed linewidths are used as input parameters in the theoretical spectra simulation, which is also improved by the inclusion of an interbranch interference with coherent Stokes Raman scattering (CSRS). These theoretical spectra are used to evaluate the temperature from experimental CARS spectra of acetylene-nitrogen mixtures recorded in the pressure range 0.1-0.6 MPa. The new linewidths (NLWs) and the CSRS interference inclusion result in a clear improvement of spectral fitting as well as in reasonable values of evaluated temperatures. Copyright (c) 2006 John Wiley & Sons, Ltd.
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