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Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

BACKGROUND: This study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia. Qualitative interviews lasting up to 90 min were conducted i...

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Detalles Bibliográficos
Autores principales: Aldhouse, Natalie V. J., Kitchen, Helen, Johnson, Chloe, Marshall, Chris, Pegram, Hannah, Pease, Sheryl, Collins, Sam, Baker, Christine L., Beaverson, Katherine, Crews, Chandler, Massey, Jill, Wyrwich, Kathleen W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9077640/
https://www.ncbi.nlm.nih.gov/pubmed/35525989
http://dx.doi.org/10.1186/s13023-022-02333-6