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.
European regulators’ views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials. Haberkamp M, Moseley J, Athanasiou D, de Andres-Trelles F, Elferink A, Rosa MM, Magrelli A. Neuromuscul Disord. 2019 Jun 7. pii: S0960-8966(18)31309-9. doi: 10.1016/j.nmd.2019.06.003. [Epub ahead of print]