Evidence map›Paper›PMID 39973462›Full record

ArticleJournal of neuromuscular diseases2024

Gross motor delays in infants and young boys with Duchenne muscular dystrophy.

Linda P Lowes, Natalie F Reash, Megan A Iammarino, Anne M Connolly, Lindsay Pietruszewski, Melissa A Smith, Jing Peng, Christopher L Steiner, Chang-Yong Tsao, Megan A Waldrop and 5 more

Abstract read
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In one paragraph

Article in Journal of neuromuscular diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Trial
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

15 authors.

Linda P LowesCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Natalie F ReashCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Megan A IammarinoCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Anne M ConnollyCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Lindsay PietruszewskiCenter for Cerebral Palsy, University of California Los Angeles, Los Angeles, California, USA.
Melissa A SmithCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Jing PengCenter for Biostatistics, Department of Biomedical Informatics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Christopher L SteinerCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Chang-Yong TsaoDepartment of Pediatrics, The College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Megan A WaldropCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Kevin M FlaniganCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Shannon ChagatDepartment of Neurology, Neuromuscular Division, Nationwide Children's Hospital, Columbus, Ohio, USA.
Alayne P MeyerDivision of Genetic and Genomic Medicine, Nationwide Children's Hospital, Columbus, Ohio, USA.
Jerry R MendellCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Lindsay N AlfanoCenter for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD), an X-linked progressive neurodegenerative disorder, is being added to required universal screening programs for newborns in the United States. It is estimated that this will result in around 880 patients presenting at clinics in infancy. Very little is known about the early gross motor abilities in infants and young boys with DMD.

objectiveDescribe the early gross motor skill acquisition in boys in our clinic with DMD.

methodsBetween the years 2016 and 2024, 90 boys with DMD under the age of 6 years (2 months - 5 years 10 months at their first visit) were evaluated as part of their standard of care during regularly scheduled clinic visits using the using the Bayley Scales of Infant & Toddler Development, Third Edition (Bayley-III). Forty-seven boys were seen in clinic longitudinally with two to six follow up visits for a total of 129 assessments.

resultsNinety four percent of the boys with DMD seen in our clinic demonstrated delays in gross motor skills across the age span when compared with normative controls. None of the boys reached a ceiling on the Bayley-III despite being older than the intended age range of the test.

conclusionsOur clinic data showed an almost universal gross motor delay in infants and toddlers that did not diminish over time.

Indexed as

Child DevelopmentDevelopmental DisabilitiesMotor SkillsMotor Skills DisordersMuscular Dystrophy, DuchenneChild, PreschoolHumansInfantLongitudinal StudiesMalechild developmentdevelopmental delay disordersDuchenne muscular dystrophygrowth and developmentinfant developmentmotor skillsmuscular dystrophy

Identifiers

PMID39973462
PMCPMC13108003

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Registered trials

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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.