Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation
Year: 2025
Type: article
Study design: Observational Study
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.
Authors: Francesco Muntoni, James Signorovitch, Michaela Johnson, Andres Gomez-Lievano, Nate Posner, Patricia Dorling, Katherine Beaverson, Jose Alvir, Matthias Mahn, Susan J Ward, Nathalie Goemans, Krista Vandenborne, Eugenio Mercuri, Craig M McDonald
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W4414434223