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Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases-A secondary analysis of a randomized clinical trial.

<h4>Background</h4>In clinical research, the most appropriate way to assess the effect of an intervention is to conduct a randomized controlled trial (RCT). In the field of rare diseases, conducting an RCT is challenging, resulting in a low rate of clinical trials, with a high frequency of early ter...

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Principais autores: Stephanie Wied, Ralf-Dieter Hilgers, Nicole Heussen, Katarzyna Kotulska, Maya Dirani, Mathieu Kuchenbuch, Sergiusz Jozwiak, Rima Nabbout
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science (PLoS) 2024-01-01
coleção:PLoS ONE
Acesso em linha:https://doi.org/10.1371/journal.pone.0312936
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