First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases.

Laurent Servais, Eric Camino, Aude Clement,Craig M McDonald,Jacek Lukawy,Linda P Lowes,Damien Eggenspieler,Francesca Cerreta,Paul Strijbos

Digital biomarkers（2021）

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摘要

Focusing on SV95C as a key example, we describe perspectives of multiple stakeholders on the promise of novel digital endpoints in neuromuscular disease drug development.

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关键词

Digital endpoints,Duchenne muscular dystrophy,Neuromuscular disease

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