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A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children
Sponsor: West China Second University Hospital
Summary
This is a prospective, multicenter, longitudinal observational cohort study aimed at understanding the progression of Duchenne Muscular Dystrophy (DMD). The primary objective is to identify and integrate key biomarkers from multiple sources-including motor function assessments, body composition (muscle and fat distribution), clinical laboratory tests, and cardiopulmonary imaging-to delineate comprehensive disease trajectories. By analyzing how these factors change over time in a large cohort, the study seeks to develop a robust model that can identify patterns of disease progression. The ultimate goal is to generate evidence that may aid in forecasting individual patient outcomes and inform the future development of personalized rehabilitation and therapeutic strategies.
Official title: A Real-World, Multicenter Cohort Study on the Natural History of Duchenne and Becker Muscular Dystrophy in Children From Western China
Key Details
Gender
MALE
Age Range
1 Year - 18 Years
Study Type
OBSERVATIONAL
Enrollment
500
Start Date
2026-04-20
Completion Date
2028-12-31
Last Updated
2026-04-06
Healthy Volunteers
Not specified
Conditions
Interventions
No interventions are applied; participants are followed for natural history observation only
This is an observational natural history study. No experimental interventions, treatments, or drugs are administered to participants. All data is collected through routine clinical care and follow-up assessments to document the natural progression of Duchenne and Becker Muscular Dystrophy.
Locations (1)
West China Second University Hospital, Sichuan University
Chengdu, China