08/11/2018
Documentación
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…Recruitment of pediatric patients in randomized clinical trials is hampered by the rarity of many conditions and by ethical constraints. The objective of this paper is to give an overview of design options to obtain a statistically valid result while including a minimum number of subjects.Overview and discussion of several approaches to conduct valid randomized […]
…Clinical trials to evaluate patients affected by rare diseases are often hampered by the difficulty of recruiting a critical sample size. Registries for rare conditions are thus extremely powerful tools for overcoming recruitment problems. Here we present and discuss the international experience with alpha1-antitrypsin deficiency achieved by the Alpha One International Registry, and national experience […]
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