Ultrasound Study to Monitor Muscle Health in Boys and Men with Duchenne and Becker Muscular Dystrophies

What this Trial Is About

Researchers are evaluating a multiparametric ultrasound approach to non-invasively monitor disease progression in boys and men with Duchenne and Becker Muscular Dystrophies. This study aims to identify objective markers of disease progression by comparing affected individuals with healthy age-matched controls over a 12-month period. Duchenne and Becker muscular dystrophies are progressive muscle-wasting diseases that cause severe muscle weakness and functional impairments, with Duchenne typically diagnosed around ages three to four and Becker showing a later onset and slower progression. Participants include ambulatory and non-ambulatory males with Duchenne Muscular Dystrophy aged 5 to 30 years, males with Becker Muscular Dystrophy aged 5 to 60 years, and healthy age-matched male controls aged 5 to 60 years. The study uses advanced ultrasound technologies such as shear wave elastography, 3D freehand ultrasound, and power Doppler to assess muscle structure, mechanics, fat content, fibrosis, inflammation, and blood flow. These measurements will be related to muscle strength and daily activity performance to develop sensitive imaging biomarkers for muscular dystrophy progression. Participants will undergo baseline and 12-month follow-up ultrasound assessments of their muscles, along with evaluations of muscle function and daily activities. The study will monitor changes over time to understand disease progression and validate ultrasound as a surrogate outcome measure for future clinical trials. This observational study involves no treatment, focusing on imaging and functional assessments over one year, with data collected at the start and end of the study period.

Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies