First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD.
作者信息
Servais Laurent, Eggenspieler Damien, Poleur Margaux, Grelet Marc, Muntoni Francesco, Strijbos Paul, Annoussamy Mélanie
机构信息
Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.
Division of Child Neurology, Department of Pediatrics, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
出版信息
Nat Med. 2023 Oct;29(10):2391-2392. doi: 10.1038/s41591-023-02459-5.
PMID:37814063
Abstract
摘要
Zotero 插件