Tracking Motor and Functional Changes Over Time in People with FSHD to Improve Care and Support Drug Development

What this Trial Is About

Researchers are conducting a long-term observational study called Motor Outcomes to Validate Evaluations in FSHD MOVE FSHD to better understand how facioscapulohumeral muscular dystrophy FSHD affects motor and functional abilities over time. This study aims to improve clinical care and support drug development by tracking changes in motor function and health outcomes such as breathing and wheelchair use in people with FSHD. The study is led by the University of Kansas Medical Center and plans to follow around 450 participants for at least three years. Participants will continue to receive their usual medical care as determined by their doctors. Study visits are expected to occur at least once a year following standard care schedules. A subgroup of approximately 200 participants will take part in additional assessments including whole body MRI, reachable workspace evaluations, optional muscle biopsies, and use of wearable devices for those in the United States. During the study, researchers will collect a variety of motor and functional measures such as a 10-meter walkrun test, shoulder and arm range of motion and function, and lung function tests spirometry over a period of up to five years. Other assessments include trunk and hand function, timed up and go tests, clinical severity scores, patient-reported outcomes, muscle strength testing, and exercise and pain evaluations. Saliva samples will be collected once for methylation analysis. These measures will help track disease progression and impacts on daily activities.

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)