presented by Claudia Senesac
Duchenne Muscular Dystrophy (DMD) is the most common childhood muscular dystrophy. Much of what we have known about this disease has been based on years of clinical observation, muscle biopsy, and other limited testing. Longitudinal studies and clinical trials are shaping our “new understanding” of this disease. The courses related to DMD will span pathophysiology, research, the development of therapy recommendations, and quality of life issues. Therapy recommendations are based on predictive models of biomarkers and function helping therapists and families plan for the future. Therapists play a critical role in caring for boys and young men with this disease.
Claudia Senesac is a Clinical Associate professor at University of Florida in the Doctor of Physical Therapy department. Her teaching responsibilities include Functional Anatomy dissection and Pediatrics in Physical Therapy. She has over 37 years of pediatric clinical experience. She has been the owner and administrator of a pediatric physical therapy private practice since 1984 and is a board certified clinical specialist in Pediatrics. Claudia is still providing therapy to children with a variety of conditions. Her research focus has been in Duchenne Muscular Dystrophy (DMD). She is a sub-investigator for a multi-center NIH funded grant investigating Magnetic Resonance Imaging and Biomarkers in boys with Duchenne Muscular Dystrophy. In addition, she has spearheaded investigating the reliability and common errors seen in outcome measures in DMD across research and clinic sites. She is a national educator for Parent Project Muscular Dystrophy translating research to clinical practice. She has several publications in peer reviewed journals related to Duchenne Muscular Dystrophy as author and co-author. Most recently she was the recipient of the International Educator of the Year award for the College of Public Health and Health Professions at UF, 2016, and in 2014 Outstanding Pediatric Clinician Award from the Pediatric Section of the APTA.
The chapter will provide foundational background information to enhance the participants understanding of DMD. Signs and symptoms of this disease will be detailed and the process of differential diagnosis reviewed. The most common medications and supplements will be covered.
Identified biomarkers have helped to further our understanding of this disease and monitor disease progression. Selected biomarkers have been developed and are emerging as outcome measures in research.
Longitudinal research studies shed light on our current understanding of DMD. Correlations of multiple outcomes help make predictions for function. Predictions of function allow for planning for the future.