Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey

Senesac CR, Lott DJ, Willcocks RJ, Duong T, Smith BK. Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey. Journal of neuromuscular diseases 2019;6:75-83.

Senesac_2018_J Neuromuscul Dis. Dec 14

Assessment of outcome measures of skeletal muscle function that could be used in clinical trials. There are several functional tests that could be standardized between different trial sites.

 

Abstract

INTRODUCTION: Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by a dystrophin protein deficiency. Dystrophin functions to stabilize and protect the muscle fiber during muscle contraction; thus, the absence of functional dystrophin protein leads to muscle injury. DMD patients experience progressive muscle necrosis, loss of function, and ultimately succumb to respiratory failure or cardiomyopathy. Exercise is known to improve muscle health and strength in healthy individuals as well as positively affect other systems. Because of this, exercise has been investigated as a potential therapeutic approach for DMD.

METHODS: This review aims to provide a concise presentation of the exercise literature with a focus on dystrophin-deficient muscle. Our intent was to identify trends and gaps in knowledge with an appreciation of exercise modality.

RESULTS: After compiling data from mouse and human studies, it became apparent that endurance exercises such as a swimming and voluntary wheel running have therapeutic potential in limb muscles of mice and respiratory training was beneficial in humans. However, in the comparatively few long-term investigations, the effect of low-intensity training on cardiac and respiratory muscles was contradictory. In addition, the effect of exercise on other systems is largely unknown.

CONCLUSIONS: To safely prescribe exercise as a therapy to DMD patients, multisystemic investigations are needed including the evaluation of respiratory and cardiac muscle.

 

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