نتایج جستجو برای: rare
تعداد نتایج: 240739 فیلتر نتایج به سال:
“Nature is nowhere accustomed more openly to display her secret mysteries than in cases where she shows traces of her workings apart from the beaten path; nor is there any better way to advance the proper practice of medicine than to give our minds to the discovery of the usual law of nature, by the careful investigation of cases of rarer forms of disease. For it has been found in almost all th...
I study the impact of the Orphan Drug Act (ODA), which established tax incentives for rare disease drug development. I examine the flow of new clinical drug trials for a large set of rare diseases. Among more prevalent rare diseases, the ODA led to a significant and sustained increase in new trials. The impact for less prevalent rare diseases was limited to an increase in the stock of drugs. Ta...
Introduction Conclusions References
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We present here by direct argument the classical Clopper-Pearson (1934) “exact” confidence bounds and corresponding intervals for the parameter p of the binomial distribution. The same arguments can be applied to derive confidence bounds and intervals for the negative binomial parameter p, for the Poisson parameter λ, for the ratio of two Poisson parameters, ρ = λ1/λ2, and for the parameter D o...
Scientific, technical and medical advances continue to raise consequential ethical questions and dilemmas also in the field of rare diseases. Difficult and complex issues of medical ethics in rare diseases are presented and several different ethical problems, like those regarding inborn errors of metabolism, are discussed.
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