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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...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer International Publishing
2023
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| Materias: | |
| 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 |