A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

Study 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.

Eligibility

Primary outcome measure(s)

• NorthStar Ambulatory Assessment (NSAA) score — Baseline and 24 months
  The NorthStar Ambulatory Assessment (NSAA) is a validated clinical scale to measure motor function in ambulatory boys with Duchenne or Becker Muscular Dystrophy.

Trial sites (1)

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