Study of Extracellular RNA as Biomarkers in Duchenne and Becker Muscular Dystrophy Using Urine and Blood Samples for Less Invasive Testing

What this Trial Is About

Researchers are investigating less invasive ways to measure the activity and severity of Duchenne Muscular Dystrophy DMD and Becker Muscular Dystrophy BMD by studying extracellular RNA in urine and blood samples. Currently, muscle biopsies are used, which are more invasive. This observational study aims to explore whether analyzing urine and blood can provide useful information instead. The study is sponsored by Massachusetts General Hospital and focuses on improving how these muscular dystrophies are monitored. Participants will provide a single urine sample and have one blood draw for study purposes. There are no additional treatments or interventions involved, as this is an observational study. The samples will be examined to identify extracellular RNA biomarkers that could reflect disease activity or severity. During the study, participants will only need to provide these samples once. Researchers will analyze the extracellular RNA in these biofluids over a period of 4 years to understand their potential as biomarkers. The study involves males aged 5 years and older with DMD or BMD, and control males and females aged 18 years and older without muscular dystrophy. There is no long-term treatment or extended follow-up required beyond the sample collection.

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy