Journal
MUSCLE & NERVE
Volume 48, Issue 1, Pages 32-54Publisher
WILEY
DOI: 10.1002/mus.23807
Keywords
adolescent; adult; child; preschool; follow-up study; health status; human; locomotion; male; muscle strength; physiology; muscular dystrophies; classification; muscular dystrophies; Duchenne; physiopathology; muscular dystrophies; therapy; phenotype; quality of life; psychology; respiratory function test
Categories
Funding
- U.S. Department of Education/NIDRR [H133B031118, H133B090001]
- U.S. Department of Defense [W81XWH-09-1-0592]
- National Institutes of Health [UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875, RO1AR062380]
- Parent Project Muscular Dystrophy
Ask authors/readers for more resources
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. Methods The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments. Results Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those 6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years). Conclusions Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics. Muscle Nerve, 2013
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available