🌐 EnglishEN

AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

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

RecruitingNAAges 4 Months - anyAllCentre Hospitalier Universitaire de Liege
NCT07286565 on ClinicalTrials.gov ↗
💬

In plain words

What the trial is, and whether it could suit you

The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) identified through newborn screening, family testing, or incidental diagnosis. The study will enroll 100 children and follow them longitudinally for up to 30 months. Participants are remotely recruited, and all procedures, including consent, questionnaires, and follow-up visits, are conducted by phone or video conferencing without any hospital visits. Children will use age-appropriate wearable devices at home: MAIJU®, a sensorized garment for non-ambulant infants, and Syde®, an ankle-worn sensor for ambulant children. Data collection includes digital motor endpoints, clinical information, and quality of life (PedsQL). Primary objectives are to validate digital

Sign in to get the eligibility rules as simple questions to take to your doctor.

Sign in
📋 Eligibility rules (as published)
Inclusion Criteria: Genetically confirmed SMA and avalaible MSNA2 copy number: * Identified by newborn screening, * Identified by family screening, or incidental diagnosis in pre-symptomatic stage * Treated (or follow-up possible for patients with 4 SMN2 copies) Genetically confirmed DMD: * Identified by newborn screening, * Identified by family screening, or incidental diagnosis in pre-symptomatic stage * Age \< 4 years at inclusion Legal guardian able to provide informed consent Exclusion Criteria: * Any acute or chronic condition that, in the investigator's opinion, significantly interferes with assessments and/or motor development. * Participation in a therapeutic trial. * Lack of internet connection.
💊

What it tests

  • MAIJU
  • Syde
  • Questionnaires
  • PedsQL Questionnaire
📍

Where

1 site
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy