Personalized Strength and Balance Exercise Training for Adults Aged 18 to 70 With Rare Neuromuscular Disorders A Multi-center, Randomized Study to Improve Physical Function and Dynamic Balance

What this Trial Is About

Researchers are evaluating the effects of personalized exercise treatment on dynamic balance and physical function compared to regular follow-up care in adults with rare neuromuscular disorders, specifically Charcot-Marie-Tooth CMT, Facioscapulohumeral Muscular Dystrophy FSHD, and Myotonic Dystrophy Type 1 DM1. The study aims to see if the exercise intervention group experiences greater improvements in dynamic balance and long-term benefits compared to the control group receiving usual care. Participants in the experimental group will undergo a four-month exercise program that starts with a 12-day personalized exercise regimen at a rehabilitation center, followed by a three-month digital intervention, and then a second 12-day personalized exercise program at the rehabilitation center. The control group will continue with their usual care, which includes hospital and community health system appointments as recommended individually. During the study, participants will be assessed at baseline, 4 months, 10 months, and 16 months for dynamic balance using the Mini-Balance Evaluation Systems Test Mini-BESTest and other measures such as fatigue severity, strength and function, metabolomics, body composition via Dual Energy X-ray Absorptiometry, neuromuscular ultrasound, physical activity, motor unit number estimation, and health-related quality of life questionnaires. The study involves 120 individuals and includes long-term follow-up for the intervention group to monitor sustained effects.

Personalized Training for People With Rare Neuromuscular Disorders