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The cooperative int...
The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures
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Henricson, E. K. (author)
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Abresch, R. T. (author)
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Cnaan, A. (author)
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Hu, F. M. (author)
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Duong, T. (author)
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Arrieta, A. (author)
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Han, J. (author)
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Escolar, D. M. (author)
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Florence, J. M. (author)
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Clemens, P. R. (author)
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Hoffman, E. P. (author)
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McDonald, C. M. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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(creator_code:org_t)
- 2013-05-06
- 2013
- English.
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In: Muscle & Nerve. - : Wiley. - 0148-639X. ; 48:1, s. 55-67
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Abstract
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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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
Keyword
- adolescent
- adult
- child
- preschool
- follow-up studies
- health status
- humans
- locomotion
- male
- 6-minute walk test
- star ambulatory assessment
- muscular-dystrophy
- prednisone therapy
- reliability
- boys
- profiles
- strength
- children
- protocol
Publication and Content Type
- ref (subject category)
- art (subject category)
Find in a library
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- By the author/editor
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Henricson, E. K.
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Abresch, R. T.
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Cnaan, A.
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Hu, F. M.
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Duong, T.
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Arrieta, A.
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show more...
-
Han, J.
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Escolar, D. M.
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Florence, J. M.
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Clemens, P. R.
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Hoffman, E. P.
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McDonald, C. M.
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Tulinius, Mar, 1 ...
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Neurology
- Articles in the publication
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Muscle & Nerve
- By the university
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University of Gothenburg