Evidence map›Paper›PMID 41760395›Full record

ArticleJournal of neurology, neurosurgery, and psychiatry2026

Respiratory function in Becker muscular dystrophy: a comprehensive longitudinal study.

Pietro Riguzzi, Emma Grover, Marianela Schiava, Meredith K James, Jordi Diaz-Manera, Giorgio Tasca, John Bourke, Holly Borland, Dionne Moat, Maha Elseed and 17 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of neurology, neurosurgery, and psychiatry, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

27 authors.

Pietro RiguzziTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.ORCID http://orcid.org/0009-0006-0241-2174
Emma GroverTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Marianela SchiavaTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.ORCID http://orcid.org/0000-0002-2709-265X
Meredith K JamesTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Jordi Diaz-ManeraTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.ORCID http://orcid.org/0000-0003-2941-7988
Giorgio TascaTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
John BourkeTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Holly BorlandTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Dionne MoatTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Maha ElseedTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Jassi Michell-SodhiTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Carla Bolaño-DíazTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Karen WongTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Ariele Barreto HaagsmaTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Emma RobinsonTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Tara ReevesTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Peter WaldockTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Elizabeth HarrisTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Michelle McCallumTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Giulio GadaletaTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.ORCID http://orcid.org/0000-0001-6563-8222
Elena PegoraroDepartment of Neurosciences DNS, Universita degli Studi di Padova, Padua, Italy.ORCID http://orcid.org/0000-0002-7740-4156
Luca BelloDepartment of Neurosciences DNS, Universita degli Studi di Padova, Padua, Italy.ORCID http://orcid.org/0000-0002-3075-6525
Ben MesserNorth East Assisted Ventilation Service, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Robert Muni-LofraTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Volker StraubTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Chiara Marini BettoloTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK.
Michela GuglieriTranslational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne, UK michela.guglieri@newcastle.ac.uk.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBecker muscular dystrophy (BMD) is a rare X-linked neuromuscular disorder predominantly affecting males. While respiratory function has been characterised in small cohorts over limited time frames, comprehensive long-term longitudinal data remain lacking, as do established care guidelines to inform respiratory surveillance in BMD.

methodsIn this retrospective analysis, we present a large longitudinal analysis of respiratory function in patients with BMD, based on 1360 spirometry measurements from a single-centre cohort of 152 patients. Analysed assessments spanned paediatric to adult ages (3.4-86.3 years), with a mean follow-up duration of 11 years per patient.Linear mixed-effects models were used to study longitudinal changes in respiratory function.

resultsRespiratory decline in BMD appears gradual and variable, typically not affecting children (<18 years of age).Respiratory support was infrequent (11.2%), always non-invasive and limited to nocturnal use.Loss of ambulation emerged as a strong predictor of faster decline in forced vital capacity percentage of predicted (FVC%) (estimate -0.58%/year, p=0.002), with the requirement for assistive walking devices marking a critical transitional stage.Upper limb function, assessed via the PUL 2.0 entry item, correlated significantly with FVC%, particularly among non-ambulant patients (rho=0.60, p=0.02). Cardiac involvement showed a limited effect on respiratory function, likely driven by patients with more advanced cardiomyopathy.No consistent genotype-phenotype correlations were observed.

conclusionThese findings provide important evidence to inform clinical management, supporting the recommendation of individualised respiratory monitoring strategies and contributing to the design and interpretation of clinical trials in BMD.

Indexed as

Muscular Dystrophy, DuchenneAdolescentAdultAgedChildChild, PreschoolFemaleHumansLongitudinal StudiesMaleMiddle AgedRespiratory Function TestsRetrospective StudiesSpirometryVital CapacityYoung AdultDYSTROPHINMUSCULAR DYSTROPHYMYOPATHYNEUROMUSCULARRESPIRATORY MEDICINE

Identifiers

PMID41760395
PMCPMC13151537

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.