Sma: registries, biomarkers & outcome measures

The objective is to compare the disease course of untreated and Spinraza-treated patients with spinal muscular atrophy (SMA) as assessed by continuous measure issued from a wearable device (ActiMyo Ⓡ). ActiMyoⓇ, a magneto-inertial device, permits gait and activity analysis of patients in an uncontrolled environment. The European Medical Agency qualified the 95th centile of stride velocity (SV95C), a digital outcome computed through the sensors, as a secondary endpoint in Duchenne muscular dystrophy (DMD).

Source: Neuromuscular Disorders - September 28, 2020 Category: Neurology Authors: C. Lilien, M. Annoussamy, M. Polleur, A. Seferian, O. Boespflug-Tanguy, K. Gorni, D. Eggenspieler, L. Servais Source Type: research