Journal
MUSCLE & NERVE
Volume 48, Issue 1, Pages 55-67Publisher
WILEY
DOI: 10.1002/mus.23808
Keywords
adolescent; adult; child; preschool; follow-up studies; health status; humans; locomotion; male; muscular dystrophies; classification; muscular dystrophies; Duchenne; physiopathology; muscular dystrophies; therapy; muscle strength; physiology; phenotype; quality of life; psychology; respiratory function tests
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Funding
- U.S. Department of Education/NIDRR [H133B031118, H133B090001]
- U.S. Department of Defense [W81XWH-09-1-0592]
- NIH [UL1RR031988, U54HD053177, UL1RR 024992, U54RR026139, 2U54HD053177, G12RR003051, 1R01AR061875, RO1AR062380]
- Parent Project Muscular Dystrophy
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Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional milestone scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4 +/- 0.39 MMT unit/year, compared with -0.4 +/- 0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy. Muscle Nerve, 2013
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