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Scully MA, Cwik VA, Marshall BC, et al. Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data? Neurology 2013;80:583-589. This short article describes variations in care across health regions in the USA and makes a recommendation for all centres to report their outcomes. It suggests that published guidelines are not being universally followed and makes an analogy with the situation for adults with CF in the early 1990s.
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DMD Care Considerations Working Group. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010;9:177-189.
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Schram G, Fournier A, Leduc H, et al. All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy. J Am Coll Cardiol 2013;61:948-954. This study demonstrates cardiac follow-up data for boys with DMD on long-term steroid treatment and demonstrates a significant cardio-protective effect with delayed onset of cardiomyopathy.
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Landfeldt E, Lindgren P, Bell C, et al. The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology 2014. [Epub ahead of print] This study was designed to estimate the total cost of illness and economic burden of DMD. Patients from Italy, United Kingdom, Germany and USA were included (770 in total), direct medical costs included hospital and physiotherapy appointments, tests and assessments, specialist physicians, medication, aids and devices which had an annual cost of $23 920-54 270. This cost was greatest for patients in the late nonambulant stage of the condition.
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Neurology
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Landfeldt, E.1
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