New Perspectives in the Management of Duchenne Muscular Dystrophy
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Population
Patients with Duchenne muscular dystrophy (DMD)
Design
Review
Authors
Discussion
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Member takes
Implication
May support 6MWT as primary endpoint in ambulatory DMD trials; leaves open prospective validation across therapies.
Structured PICO
P
Population
Patients with Duchenne muscular dystrophy (DMD)
I
Intervention
Multidisciplinary clinical management, corticosteroid therapy, and emerging therapies
The 6-minute walk test is a robust and clinically meaningful primary endpoint for evaluating therapies in ambulatory patients with Duchenne muscular dystrophy.
Cite This Study
Goemans et al. (2014) studied this question.
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