Dtsch Arztebl Int
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Data obtained from screened newborns and from persons at known risk for Fabry disease suggest that this condition is much more common in Germany than previously assumed. Its clinical manifestations are very diverse, and its differential diagnosis is correspondingly broad. Thus, there is often a delay before the diagnosis of Fabry disease is established. ⋯ Fabry disease is still underdiagnosed. The average delay from the onset of symptoms to diagnosis is more than a decade. Treatment with human alpha-galactosidase A produced with genetic technology can improve most of the disease's manifestations.
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Comparative Study
Confidence interval or p-value?: part 4 of a series on evaluation of scientific publications.
An understanding of p-values and confidence intervals is necessary for the evaluation of scientific articles. This article will inform the reader of the meaning and interpretation of these two statistical concepts. ⋯ P-values in scientific studies are used to determine whether a null hypothesis formulated before the performance of the study is to be accepted or rejected. In exploratory studies, p-values enable the recognition of any statistically noteworthy findings. Confidence intervals provide information about a range in which the true value lies with a certain degree of probability, as well as about the direction and strength of the demonstrated effect. This enables conclusions to be drawn about the statistical plausibility and clinical relevance of the study findings. It is often useful for both statistical measures to be reported in scientific articles, because they provide complementary types of information.