Respiratory decline in adult patients with Becker muscular dystrophy: A longitudinal study

Becker muscular dystrophy (BMD) is a rare hereditary neuromuscular disease, caused by genetic defect in the Duchenne Muscular Dystrophy (DMD) gene. We studied natural history of respiratory function and its affecting factors 23 adult BMD patients. These important data are needed for (future) clinical trials but largely lacking. Patients had median age 51 years (28–78y) follow-up duration 14 (2–25y). analysed 190 pulmonary measurements with interval one year (1–17y) measured 1.00% decline Forced Vital Capacity percent predicted (FVC%pred) per (p = 0.004). Loss ambulation significantly increased annual rate FVC was dependent patient's body mass index (BMI; p 0.015), increases BMI correlating an even more rapid deterioration FVC. A Medical Research Council (MRC) sum score correlated 0.003). conclude that patients experience significant mild decline. However, this clinically relevant after loss ambulation, which warrants vigilant subgroup.