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Updated: Feb 8, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
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Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data.

Marika Pane1, Giorgia Coratti1, Claudia Brogna1

  • 1Pediatric Neurology and Nemo Clinical Centre, Fondazione Policlinico Universitario "A. Gemelli IRCSS", Università Cattolica del Sacro Cuore, Rome, Italy.

Plos One
|June 21, 2018
PubMed
Summary
This summary is machine-generated.

This study tracked upper limb function in boys with Duchenne muscular dystrophy over 24 months. Non-ambulant boys experienced faster declines in upper limb function compared to ambulant boys.

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Area of Science:

  • Neurology
  • Pediatrics
  • Rehabilitation Medicine

Background:

  • Duchenne muscular dystrophy (DMD) is a progressive genetic disorder affecting muscle strength.
  • Upper limb function is crucial for daily activities and quality of life in individuals with DMD.
  • Longitudinal data on upper limb function changes in DMD are essential for clinical management and trial design.

Purpose of the Study:

  • To assess 24-month changes in upper limb function in ambulant and non-ambulant boys with DMD using the Performance of Upper Limb test (PUL 2.0).
  • To identify progression trajectories of upper limb function in DMD.
  • To understand factors influencing the rate of upper limb function decline.

Main Methods:

  • A cohort of 187 boys with DMD (87 ambulant, 90 non-ambulant) was studied over 24 months.
  • The revised Performance of Upper Limb test (PUL 2.0) was used to measure upper limb function.
  • Multivariate model analysis was employed to identify factors associated with changes in PUL 2.0 scores.

Main Results:

  • Significant changes in total PUL 2.0 scores were observed over time (p<0.001).
  • Non-ambulant boys had significantly lower baseline scores and a greater decrease in scores over 24 months compared to ambulant boys.
  • Entry level and ambulation status were independently associated with the rate of change in upper limb function.

Conclusions:

  • Upper limb function declines significantly over 24 months in boys with DMD, with non-ambulant individuals experiencing a more rapid decline.
  • Ambulation status and baseline functional level are key predictors of upper limb function progression in DMD.
  • These findings are valuable for clinical practice and the design of future clinical trials in DMD.