Evidence map›Paper›PMID 40172561›Full record

ArticlePhilosophical transactions. Series A, Mathematical, physical, and engineering sciences2025

Fully personalized modelling of Duchenne Muscular Dystrophy ambulation.

Victor Applebaum, Evan Baker, Thomas Kim, Georgia Stimpson, Peter Challenor, Kyle Carlton Abesser Wedgwood, Matthew Anderson, Ian Bamsey, Giovanni Baranello, Adnan Manzur and 2 more

Abstract read
In one paragraph

Article in Philosophical transactions. Series A, Mathematical, physical, and engineering sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Fully personalized modelling of Duchenne Muscular Dystrophy ambulation.Philosophical transactions. Series A, Mathematical, physical, and engineering sciences · 2025
    Article
  2. Preface to the theme issue 'Uncertainty quantification for healthcare and biological systems (Part 2)'.Philosophical transactions. Series A, Mathematical, physical, and engineering sciences · 2025
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Victor ApplebaumDepartment of Mathematics and Statistics and EPSRC Hub for Quantitative Modelling in Healthcare, University of Exeter, Exeter, UK.ORCID 0000-0002-4654-4965
Evan BakerDepartment of Mathematics and Statistics and EPSRC Hub for Quantitative Modelling in Healthcare, University of Exeter, Exeter, UK.ORCID 0000-0003-0945-4749
Thomas KimCertus Technology Associates Ltd, Exeter, UK.
Georgia StimpsonDubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.ORCID 0000-0003-2795-9898
Peter ChallenorDepartment of Mathematics and Statistics and EPSRC Hub for Quantitative Modelling in Healthcare, University of Exeter, Exeter, UK.ORCID 0000-0001-8661-2718
Kyle Carlton Abesser WedgwoodDepartment of Mathematics and Statistics and EPSRC Hub for Quantitative Modelling in Healthcare, University of Exeter, Exeter, UK.ORCID 0000-0002-8109-2765
Matthew AndersonCertus Technology Associates Ltd, Exeter, UK.
Ian BamseyCertus Technology Associates Ltd, Exeter, UK.
Giovanni BaranelloDubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.ORCID 0000-0003-4871-6692
Adnan ManzurDubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.
Francesco MuntoniDubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.ORCID 0000-0002-9102-5232
Krasimira Tsaneva-AtanasovaDepartment of Mathematics and Statistics and EPSRC Hub for Quantitative Modelling in Healthcare, University of Exeter, Exeter, UK.ORCID 0000-0002-6294-7051

Funding

EPSRC Hub for Quantitative Modelling in Healthcare
6 · The paper itself

Abstract

Duchenne Muscular Dystrophy is a progressive neuromuscular disorder characterized by the gradual weakening and deterioration of muscles, leading to loss of ambulation in affected individuals. This decline in mobility can be effectively assessed using the North Star Ambulatory Assessment (NSAA) scores, along with measures such as the 10-m walk time and the time taken to rise from the floor. We propose a dynamic linear model to predict the trajectories of these clinical outcomes, with a primary focus on NSAA scores. Our model aims to assist clinicians in forecasting the progression of the disease, thereby enabling more informed and personalized treatment plans for their patients. We also evaluate the effectiveness of our models in generating synthetic NSAA score datasets. We assess the performance of our modelling approach and compare the results with those of a previous study. We show that the most robust model demonstrates narrower prediction intervals and improved quantile coverage, indicating superior predictive accuracy and reliability.This article is part of the theme issue 'Uncertainty quantification for healthcare and biological systems (Part 2)'.

Indexed as

Muscular Dystrophy, DuchennePrecision MedicineWalkingDisease ProgressionHumansLinear ModelsReproducibility of ResultsDuchenne Muscular Dystrophydynamic linear modelGaussian processpatient trajectoriespersonalized medicinesynthetic data

Identifiers

PMID40172561
PMCPMC12105785

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.