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Updated: Jan 27, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy
1Laboratory of Muscle and Translational Therapeutics, Department of Physical Therapy, Duquesne University, Pittsburgh, PA 15228, USA. kostekm@duq.edu.
Abstract:
Precision medicine is being discussed and incorporated at all levels of health care and disease prevention, management, and treatment. Key components include new taxonomies of disease classification, the measurement and incorporation of genetics and "omics" data, biomarkers, and health care professionals who can optimize this information for a precision approach to treatment. The study and treatment of Duchenne Muscular Dystrophy is making rapid advances in these areas in addition to rapid advances in new gene and cell-based therapies. New therapies will increase the variability in disease severity, furthering a need for a precision-based approach. An area of therapy that is rarely considered in this approach is how the physiology of muscle contractions will interact with these therapies and a precision approach. As muscle pathology improves, physical activity levels will increase, which will likely be very beneficial to some patients but likely not to all. Physical activity is likely to synergistically improve these therapies and can be used to enhance muscle health and quality of life after these therapies are delivered using the tools of precision medicine.
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Purposes
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