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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...
Autores principales: | Verbeeck, Johan, Dirani, Maya, Bauer, Johann W., Hilgers, Ralf-Dieter, Molenberghs, Geert, Nabbout, Rima |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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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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