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Composite endpoints, including patient reported outcomes, in rare diseases

BACKGROUND: When assessing the efficacy of a treatment in any clinical trial, it is recommended by the International Conference on Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multiface...

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Detalles Bibliográficos
Autores principales: Verbeeck, Johan, Dirani, Maya, Bauer, Johann W., Hilgers, Ralf-Dieter, Molenberghs, Geert, Nabbout, Rima
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10474650/
https://www.ncbi.nlm.nih.gov/pubmed/37658423
http://dx.doi.org/10.1186/s13023-023-02819-x

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