Haberkamp M, Moseley J, Athanasiou D, et al. European regulators’ views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials. Neuromuscular Disord 2019;29:514-516
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 . 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.