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.
Male participants with genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD)
Age range: 1 to 18 years old (adjust to your actual age limit)
Ability to complete study assessments and follow-up visits
Participants or legal guardians provide written informed consent
Participants with other neuromuscular disorders that may confound natural history data
Participation in another interventional clinical trial that could affect disease progression
Severe comorbidities that prevent completion of study assessments
Inability to provide informed consent or comply with study procedures
This is a multicenter observational natural history study of male children and adolescents with genetically confirmed Duchenne or Becker Muscular Dystrophy. Participants will be followed longitudinally to document the natural progression of muscle weakness, functional decline, and associated comorbidities. The study population includes individuals across a range of disease severity and ages, from early childhood to late adolescence.
Cohort
Prospective
No interventions are applied; participants are followed for natural history observation only
Other interventionThis 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.
NorthStar Ambulatory Assessment (NSAA) score
The NorthStar Ambulatory Assessment (NSAA) is a validated clinical scale to measure motor function in ambulatory boys with Duchenne or Becker Muscular Dystrophy.
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This trial is not recruiting at the moment. You can still explore other options:
