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

202176 citationsReviewgold Open Access

Authors

Laurent Servais · University of Oxford

Eric Camino · Parent Project Muscular Dystrophy

Aude Clement · Roche (Switzerland)

Craig M. McDonald · University of California, Davis

Jacek Lukawy · Roche (Switzerland)

Linda Lowes · The Ohio State University

Damien Eggenspieler ·

Abstract

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.

Topics & Keywords

Muscle Physiology and Disorders Cardiomyopathy and Myosin Studies Muscle activation and electromyography studies

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Digital Biomarkers

Volume 5, Issue 2, pp. 183-190

DOI: 10.1159/000517411

Field-Weighted Citation Impact: 4.64