Strategy for Generating Blinded Evidence for Single-Arm Trials with External Controls Using Expert Review of Home Video

INTRODUCTION: Neurodegenerative diseases cause developmental delays and loss of milestones in infants and children. However, scalable outcome measures that quantify features meaningful to parents/caregivers (P/CGs) and have regulatory precedence are lacking for assessing the effectiveness of treatme...

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Detalles Bibliográficos
Autores principales: Zhang, Xinruo, Brind’Amour, Katherine, King, Kelly E., Hartmaier, Susan, Harris, Katherine, Weinstein, David A., Girman, Cynthia J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10579152/
https://www.ncbi.nlm.nih.gov/pubmed/37592153
http://dx.doi.org/10.1007/s43441-023-00568-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10579152/
https://www.ncbi.nlm.nih.gov/pubmed/37592153
http://dx.doi.org/10.1007/s43441-023-00568-4

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