European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials

Marion Haberkamp, Jane Moseley, Dimitrios Athanasiou, Fernando de Andres-Trelles, André Elferink, Mário Miguel Rosa, Armando Magrelli

Research output: Contribution to journalArticle

Abstract

Development of novel therapies for Duchenne muscular dystrophy (DMD) are driving the need for more efficient ways of detecting changes in disease- progression in DMD [1]. However, medicines' approval must be based on outcome measures that are acceptable from a regulatory perspective. In this article, European regulators provide an update on the recent regulatory consideration of a new endpoint (Stride Velocity 95th Centile (SV95C)) that could be used in therapeutic DMD trials. This new endpoint aims to quantify a patient's ambulation directly, reliably and continuously in a home environment with a wearable device.

Original languageEnglish
Pages (from-to)514-516
Number of pages3
JournalNeuromuscular Disorders
Volume29
Issue number7
DOIs
Publication statusPublished - Jul 2019

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