Digital Monitoring of Upper Limb Function in Non-Ambulant DMD

Feasibility, Reliability, Clinical Validity and Sensitivity of Digital Outcomes to Monitor Upper Limb Function in Non-ambulant Patients With Genetically Confirmed Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration. Symptoms typically begin in early childhood and result in loss of ambulation by early adolescence, followed by cardiorespiratory complications. Although early treatment, including corticosteroids and emerging therapies, can slow disease progression, sensitive tools to monitor functional decline-particularly in non-ambulant patients-remain limited.

Current assessments rely primarily on clinical scales and hospital-based evaluations, which may not detect subtle changes or reflect real-life function. Digital outcome measures derived from wearable sensors offer a promising approach for continuous, objective monitoring in daily life. This study aims to evaluate the feasibility, reliability, clinical validity, and sensitivity of digital measures to assess upper limb function in non-ambulant patients with genetically confirmed DMD. The Syde device, previously validated in ambulant DMD patients, will be investigated for its applicability in this population.

Inclusion Criteria:

• Patient with genetically confirmed Duchenne Muscular Dystrophy (DMD).
• Non-ambulant at the time of inclusion (not able to walk 10m without external aid).
• A legal guardian willing and able to provide written informed consent for participation in the study if < 18 years old.

Exclusion Criteria:

• Any acute or chronic condition that, in the opinion of the investigator, may significantly interfere with the assessments and/or motor function progression.
• Participation in an interventional clinical trial.
• No access to internet connection or alternatively no capacity to come on-site to bring the Syde every 6 months after the recording periods for data retrieval by Liège team
• Scoliosis surgery within the previous 6 months or planned within the next year