How do you assess a patient too young to follow instructions?
That’s the challenge our Program Director, Damien Eggenspieler, tackled on the ground at ICNMD 2026, presenting 18 months of data from the ActiLiège-Next natural history study.
The ActiLiège-Next Study
ActiLiège-Next is an 18-month longitudinal natural history study measuring real-world motor function in patients with Duchenne Muscular Dystrophy (DMD) under 4 years old, using the Syde wearable sensor.
Toddlers do not reliably follow instructions, and most in-clinic tests assume they do. That’s why researchers have been looking for a way to measure function without needing a child’s cooperation.
Stride Velocity 95th Centile, a passive, wearable-derived endpoint that captures ambulatory function as children simply go about their day, with no test to perform and nothing for a young patient to understand or comply with.
What the Data Revealed
- ~100% Adherence: Toddlers actually wear the sensors. Compliance held near 100% at 18 months, proof the passive design works in practice.
- >0.9: ICC: Excellent reliability. An intraclass correlation coefficient above 0.9 confirms the sensor data is consistent and trustworthy enough for clinical use.
- Clear Differentiation: SV95C objectively distinguishes young DMD patients from age-matched healthy controls, even at a very young age when the difference might not be apparent to a trained clinician’s eye.
- Sensitivity to Treatment Efficacy: When patients started steroid therapy, SV95C captured a significant improvement in the evolution slope, proof the endpoint responds to real clinical change.
Why It Matters
In DMD, early intervention can be the difference between preserving and losing motor function. These findings support SV95C as a promising, passive, digital endpoint for children under 4, paving the way for earlier, more accurate clinical trials, without burdensome tests for these young patients.
