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Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
Francesco Muntoni A, James Signorovitch, Gautam Sajeev, Nathalie Goemans, Brenda L. Y. Wong, Cuixia Tian, +12 more
40citations -
Construction and assessment of prediction rules for binary outcome in the presence of missing predictor data using multiple imputation and cross‐validation: Methodological approach and data‐based evaluation20citations
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Data from an International Multi-Centre Study of Statistics and Mathematics Anxieties and Related Variables in University Students (the SMARVUS Dataset)2023 Journal of Open Psychology Data article Mathematics Statistics Education and Methodologies Open access
Jenny Terry, Robert Malcolm Ross, Tamás Nagy, Mauricio Salgado, Patricia Garrido‐Vásquez, Jacob Owusu Sarfo, +93 more
14citations -
Covariate-specific ROC curve analysis can accommodate differences between covariate subgroups in the evaluation of diagnostic accuracy2023 Journal of Clinical Epidemiology article Mathematics Statistical Methods in Epidemiology Open access
Jenny Lee, Nick van Es, Toshihiko Takada, Frederikus Albertus Klok, Geert‐Jan Geersing, Jeffrey D. Blume, +1 more
25citations -
Handling missing values in patient-reported outcome data in the presence of intercurrent events2025 BMC Medical Research Methodology article Mathematics Statistical Methods and Bayesian Inference Open access
Doranne Thomassen, Satrajit Roychoudhury, Cecilie Delphin Amdal, Dries Reynders, Jammbe Z. Musoro, Willi Sauerbrei, +31 more
12citations -
Dose escalations in phase I studies: Feasibility of interpreting blinded pharmacodynamic data2022 British Journal of Clinical Pharmacology article Mathematics Statistical Methods in Clinical Trials Open access
Gerardus J. Hassing, Michiel Joost van Esdonk, Gerard J. P. van Westen, Adam F. Cohen, Jacobus Burggraaf, Pim Gal
2citations -
Information about missing patient-reported outcome data in breast cancer trials is frequently not documented: a scoping review2023 Journal of Clinical Epidemiology article Mathematics Statistical Methods in Clinical Trials Open access
Daniela Krepper, Johannes Maria Giesinger, Linda Dirven, Fabio Efficace, Caroline Lopes Martini, Anna Margarete Maria Thurner, +3 more
12citations -
High-dimensional, outcome-dependent missing data problems: Models for the human KIR loci2025 Statistical Methods in Medical Research article Mathematics Statistical Methods and Bayesian Inference Open access
Lars L. J. van der Burg, Hein Putter, Henning Baldauf, Jürgen Sauter, Johannes Schetelig, Liesbeth C. de Wreede, +1 more
0citations -
Analyzing Coarsened and Missing Data by Imputation Methods2025 Statistics in Medicine article Mathematics Statistical Methods and Bayesian Inference Open access
Lars L. J. van der Burg, Stefan Böhringer, Jonathan William Bartlett, Tjalling Bosse, Nanda Horeweg, Liesbeth C. de Wreede, +1 more
0citations