Using Quantitative Muscle Imaging to Study Disease Progression in Adults with Myotonic Dystrophy Ages 18 to 65

What this Trial Is About

Myotonic dystrophy DM is the most common type of muscular dystrophy in adults, causing progressive muscle weakness, myotonia, and affecting multiple body systems. This condition leads to severe disability and significantly impacts patients quality of life. Currently, there are no effective treatments that change the disease course, so researchers are exploring new ways to better understand and monitor DM progression using non-invasive methods. This research evaluates the use of Quantitative Magnetic Resonance Imaging qMRI as a non-invasive biomarker to study muscle structure and disease severity in people with DM. Traditional muscle biopsies to assess disease have been invasive and inefficient for long-term monitoring. The study compares muscle imaging findings with clinical signs and RNA splicing outcomes in DM patients, aiming to establish qMRI as a useful tool for future clinical trials and drug development. Participants include adults aged 18 to 65 with DM or healthy controls. They will undergo various muscle function tests, strength measurements, and physical performance assessments, along with MRI scans to measure muscle volume and fat content. Researchers will analyze these data to understand muscle changes and their relationship to disease severity. The study will monitor participants at baseline to collect this detailed information, contributing to improved disease assessment methods in DM.

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy