The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease of muscle characterized by a progressive loss of functional muscle mass, which is replaced with fibrofatty tissue. Historically, boys with DMD lose the ability to walk between the ages of 8-12 years, due to progressive weakness of the quadriceps coupled with the development of contractures at the hip, knee and ankle. This progressive loss in function necessitates individuals with DMD to spend less time walking and more time in wheelchairs, leading to the development of spinal deformities. Recently, corticosteroids have been shown to reduce the expected loss of muscle strength, extend the time that ambulation and standing are maintained, and minimize or eliminate spinal deformity in individuals with DMD; yet, the side effects of such treatment preclude use in some patients. To date, differences in gait patterns and other markers of disease progression between boys on corticosteroids and those not utilizing such treatment have not been objectively quantified. This lack of knowledge is a major obstacle to determining the most effective treatment for subsets of boys with DMD.
Study Type
OBSERVATIONAL
Enrollment
85
UCLA Department of Orthopaedic Surgery
Los Angeles, California, United States
Shriners Hospitals for Children
Sacramento, California, United States
Shriners Hospitals for Children
Portland, Oregon, United States
Gait pattern
computerized assessment of walking
Time frame: every six months (2x/year)
muscle strength
quantitative assessment of strength with a Biodex
Time frame: every six months (2x/year)
energy cost of walking
assessment of how much energy it takes to walk, assessed with a Cosmed K4b2
Time frame: every six months (2x/year)
gross motor functional skills
assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs
Time frame: every six months (2x/year)
Step activity Monitor-participation
measurement of the number of steps taken in the community/home environment during weekdays and weekends
Time frame: one week every six months
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