Evaluating Cardiopulmonary Exercise Testing in Duchenne Muscular Dystrophy

2013

Progressive muscle weakness beginning in the first decade of a childhood is well described in Duchenne Muscular Dystrophy (DMD). A number of interventions are in early clinical trials including genetic modifications to increase dystrophin withing skeletal muscle. Tests of global function such as the 6 minute walk test have been standardized for use as clinical trial outcome measures in boys with DMD but are a composite value representing capacity of 3 physiologic domains: cardiac, pulmonary, and skeletal muscle function. The goals of this pilot study are to demonstrate feasibility of cardiopulmunary exercise testing in boys with DMD. We will develop an adaptive protocols that may be employed in ambulatory and non ambulatory boys with physical limitations. We will determine accuracy of data obtained during cardipulmonary exercise testing as a quantitative tool for measurement by comparing with other common clinical measures.

Duchenne muscular dystrophy, exercise, testing

Performing Research or Evaluation

Health-Related Activities

Family Members/Caregivers, Children/Adolescents with Disabilities/SHCN

Specific Groups, Other

Mulit-County

Federal

N/A