Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation

We described ambulatory Duchenne muscular dystrophy (DMD) progression, across multiple functional measures, via previously established prognostic groups for loss of ambulation (LoA) and health states. Patients closer to vs. farther from LoA had greater declines in some measures (e.g., 6-min walk distance) and less change in others (e.g., timed rise from floor velocity) due to floor effects. Patients in the late vs. early ambulatory health state were concordantly shifted towards higher LoA risk. Findings further characterize health states and prognostic factors in ambulatory DMD and highlight the importance of multiple measures of function to fully characterize disease progression.

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Publication Details

Journal
Journal of Neuromuscular Diseases
Published
2025-09-23
DOI
https://doi.org/10.1177/22143602251364694
Primary Topic
Muscle Physiology and Disorders
Type
article
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article

Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation

Michaela Johnson, Katherine Beaverson, Krista Vandenborne, Andrés Gómez-Liévano et al.
Journal of Neuromuscular Diseases
Muscle Physiology and Disorders
article

Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation

Michaela Johnson, Katherine Beaverson, Krista Vandenborne, Andrés Gómez-Liévano, James Signorovitch, Eugenio Maria Mercuri, Nathalie Goemans, Craig M. McDonald, Patricia Dorling, José Alvir, Matthias Mahn, Francesco Muntoni, Susan Ward, Nathaniel Posner
article en

Abstract

We described ambulatory Duchenne muscular dystrophy (DMD) progression, across multiple functional measures, via previously established prognostic groups for loss of ambulation (LoA) and health states. Patients closer to vs. farther from LoA had greater declines in some measures (e.g., 6-min walk distance) and less change in others (e.g., timed rise from floor velocity) due to floor effects. Patients in the late vs. early ambulatory health state were concordantly shifted towards higher LoA risk. Findings further characterize health states and prognostic factors in ambulatory DMD and highlight the importance of multiple measures of function to fully characterize disease progression.

Journal of Neuromuscular Diseases
Università Cattolica del Sacro Cuore (IT), Pfizer (United States) (US), Great Ormond Street Hospital (GB), University of Florida (US), Centro Clinico Nemo (IT), Cambridge Collaborative (United States) (US), California State University, Sacramento (US), NIHR Great Ormond Street Hospital Biomedical Research Centre (GB), University College London (GB), Analysis Group (United States) (US), KU Leuven (BE)
Openalex Percentile: Top 77%
Muscle Physiology and Disorders
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