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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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- 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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- Stack, K. M., et al.
(författare)
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Sedimentology and Stratigraphy of the Shenandoah Formation, Western Fan, Jezero Crater, Mars
- 2024
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Ingår i: Journal of Geophysical Research: Planets. - 2169-9097. ; 129:2
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Tidskriftsartikel (refereegranskat)abstract
- Sedimentary fans are key targets of exploration on Mars because they record the history of surface aqueous activity and habitability. The sedimentary fan extending from the Neretva Vallis breach of Jezero crater's western rim is one of the Mars 2020 Perseverance rover's main exploration targets. Perseverance spent ∼250 sols exploring and collecting seven rock cores from the lower ∼25 m of sedimentary rock exposed within the fan's eastern scarp, a sequence informally named the “Shenandoah” formation. This study describes the sedimentology and stratigraphy of the Shenandoah formation at two areas, “Cape Nukshak” and “Hawksbill Gap,” including a characterization, interpretation, and depositional framework for the facies that comprise it. The five main facies of the Shenandoah formation include: laminated mudstone, laminated sandstone, low-angle cross stratified sandstone, thin-bedded granule sandstone, and thick-bedded granule-pebble sandstone and conglomerate. These facies are organized into three facies associations (FA): FA1, comprised of laminated and soft sediment-deformed sandstone interbedded with broad, unconfined coarser-grained granule and pebbly sandstone intervals; FA2, comprised predominantly of laterally extensive, soft-sediment deformed laminated, sulfate-bearing mudstone with lenses of low-angle cross-stratified and scoured sandstone; and FA3, comprised of dipping planar, thin-bedded sand-gravel couplets. The depositional model favored for the Shenandoah formation involves the transition from a sand-dominated distal alluvial fan setting (FA1) to a stable, widespread saline lake (FA2), followed by the progradation of a river delta system (FA3) into the lake basin. This sequence records the initiation of a relatively long-lived, habitable lacustrine and deltaic environment within Jezero crater.
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- Mendez, V., et al.
(författare)
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Novel hydrocarbonoclastic metal-tolerant Acinetobacter and Pseudomonas strains from Aconcagua river oil-polluted soil
- 2017
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Ingår i: Journal of Soil Science and Plant Nutrition. - 0718-9516. ; 17:4, s. 1074-1087
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Tidskriftsartikel (refereegranskat)abstract
- Bioremediation of sites polluted with petroleum hydrocarbons and heavy metals is a major challenge. The aim of this study was the isolation and characterization of hydrocarbon-degrading and heavy metal-tolerant bacteria. Sixteen hydrocarbonoclastic bacteria were isolated by enrichment from a crude oil-contaminated soil at Aconcagua river mouth, Central Chile. Most strains were cocci-shaped and exhibited circular cream-colored colonies with smooth texture. Isolates were resistant to bacitracin and penicillin, and two isolates were motile. Isolates were identified by 16S rRNA and rpoD and rpoB genes sequence analyses. Most isolates belonged to Gammaproteobacteria including Acinetobacter radioresistens (4 isolates), Acinetobacter calcoaceticus (1), Pseudomonas stutzeri (2) and Pseudomonas chloritidismutans (1). Seven isolates possessed 97% 16S rRNA gene similarity with A. calcoaceticus ATCC 23055T, suggesting that these probably represent a new Acinetobacter species. One isolate is an Actinobacteria of the Kocuria genus. All isolates were able to grow on crude oil, whereas eleven Acinetobacter and Pseudomonas strains grew on n-hexadecane. Pseudomonas isolates grew on fluorene (DM88 and DM95) and naphthalene (DD74). Acinetobacter isolates grew on fluorene (DD75, DD79 and DM81) and phenanthrene (DM82). Remarkably, most isolates (except DD79) exhibited copper or cadmium tolerance. These novel hydrocarbonoclastic and heavy metal-tolerant Pseudomonas and Acinetobacter strains are potential biocatalysts for bioremediation.
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- Valenzuela, M., et al.
(författare)
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Analyses of Virulence Genes of Clavibacter michiganensis subsp. michiganensis Strains Reveal Heterogeneity and Deletions That Correlate with Pathogenicity
- 2021
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Ingår i: Microorganisms. - : MDPI AG. - 2076-2607. ; 9:7
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Tidskriftsartikel (refereegranskat)abstract
- Clavibacter michiganensis subsp. michiganensis (Cmm) is the causal agent of bacterial canker of tomato. Differences in virulence between Cmm strains have been reported. The aim of this study was the characterization of nine Cmm strains isolated in Chile to reveal the causes of their differences in virulence. The virulence assays in tomato seedlings revealed different levels of severity associated with the strains, with two highly virulent strains and one causing only mild symptoms. The two most virulent showed increased cellulase activity, and no cellulase activity was observed in the strain causing mild symptoms. In three strains, including the two most virulent strains, PCR amplification of the 10 virulence genes analyzed was observed. In the strain causing mild symptoms, no amplification was observed for five genes, including celA. Sequence and cluster analyses of six virulence genes grouped the strains, as has been previously reported, except for gene pelA1. Gene sequence analysis from the genomes of five Chilean strains revealed the presence of deletions in the virulence genes, celB, xysA, pat-1, and phpA. The results of this study allow us to establish correlations between the differences observed in disease severity and the presence/absence of genes and deletions not previously reported.
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| 10. |
- 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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