Active-NBS Liege - Monitoring the Motor Development of Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified Through Newborn Screening

What this Trial Is About

Researchers are evaluating digital measures of motor development in children diagnosed with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) through newborn screening, family testing, or incidental diagnosis. This fully remote, observational study aims to validate digital biomarkers that can detect early motor deficits and model motor trajectories in very young children with these serious neuromuscular disorders. The study will enroll up to 100 children and follow them over a period of up to 30 months. Participants will use age-appropriate wearable devices at home according to their motor abilities. Non-ambulant infants will wear the MAIJU4 sensorized jumpsuit, while ambulant children will wear the Syde4 ankle sensor to capture precise gait data including stride velocity. Families will also complete questionnaires about medical history and quality of life (PedsQL). All recruitment, consent, and assessments are conducted remotely via phone or video calls, with no hospital visits required. Throughout the study, children will be assessed every six months using digital motor endpoints, clinical information, and quality of life measures. Data collection includes continuous recording periods lasting up to four weeks every three months and monthly one-day recordings over two years. Researchers will monitor compliance with device use and analyze walking pattern characteristics to understand motor development and differences related to genetics and treatment exposure. The study prioritizes minimal risk by using non-invasive sensors with no known side effects.

Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening