About Becker Muscular Dystrophy
Becker Muscular Dystrophy is an X-linked recessive disorder caused by in-frame mutations in the DMD gene, resulting in reduced but partially functional dystrophin protein. Unlike Duchenne MD, patients typically remain ambulatory past age 16 and the course is more variable, ranging from mild proximal weakness to severe cardiac and respiratory involvement. Cardiomyopathy is a leading cause of morbidity and mortality in BMD.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Becker Muscular Dystrophy trials.
Genetic testing confirming an in-frame DMD deletion or duplication is typically required; exon-level results from multiplex ligation-dependent probe amplification (MLPA) are preferred
Cardiac trials specifically target BMD due to cardiomyopathy prevalence — cardiac MRI with ejection fraction data is frequently a screening prerequisite
Ambulatory status and 6-minute walk distance (6MWD) are primary stratification variables; document these with a physiotherapist before applying
Patient Resources
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