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Updated: May 31, 2026
09:18
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study.
E Mazzone1, G Vasco, M P Sormani
1Department of Paediatric Neurology, Catholic University, Rome.
Neurology
|July 8, 2011
Summary
This study tracked changes in Duchenne muscular dystrophy (DMD) patients over 12 months. While most declined, younger boys showed improvements in the 6-minute walk test (6MWT) and North Star Ambulatory Assessment (NSAA).
Area of Science:
- Neurology
- Pediatrics
- Clinical Medicine
Background:
- Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder affecting ambulant boys.
- Longitudinal data on functional outcome measures are crucial for understanding disease progression and evaluating interventions.
Purpose of the Study:
- To assess changes in the 6-minute walk test (6MWT) and North Star Ambulatory Assessment (NSAA) over 12 months in ambulant boys with DMD.
- To explore the influence of age and steroid treatment on these outcome measures.
- To establish the spectrum of functional changes in DMD.
Main Methods:
- A longitudinal, multicentric cohort study involving 106 ambulant boys with DMD.
- Standardized assessments of 6MWT and NSAA at baseline and 12 months.
- Collection of clinical data including patient age and steroid treatment status.
Main Results:
- A mean decline of 25.8 meters was observed in the 6MWT and 2.2 points in the NSAA over 12 months.
- Younger boys (up to age 7) demonstrated potential for improvement in both 6MWT and NSAA.
- A significant correlation was found between NSAA and 6MWT (r = 0.52, p < 0.001).
Conclusions:
- The study provides valuable 12-month longitudinal data for the NSAA and 6MWT in ambulant DMD patients.
- These findings can inform the design and power calculations for future clinical trials in DMD.
- Understanding age-related changes is important for interpreting outcome measure results.
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