Fiche société
Centre Hospitalier Universitaire de Liege
Traitements, essais et publications liés.
Traitements2programmes
Essais1liés
Publications3liées
SourceDBlocale
Traitements
2| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| SydeDuchenne 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. | Myopathies | Non applicable | Ralentissement de la progression + Traitement symptomatique | Belgium | À vérifier |
| SydeDuchenne 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. | Myopathies | Non applicable | Ralentissement de la progression + Traitement symptomatique | Belgium | À vérifier |
Essais cliniques
1| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| Syde | Myopathies | Non applicable | NCT07664124 | Acti-nRoll — Digital Monitoring of Upper Limb Function in Non-Ambulant DMD | NOT_YET_RECRUITING |
Publications
3| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| Syde | Wearable sensors in paediatric neurology. | Developmental medicine and child neurology | ||
| Syde | Operational control mechanism and pilot-scale demonstration of integrated fixed-film activated sludge partial nitritation/anammox system. | Bioresource technology | ||
| Syde | Stride-level measurement of gait as an early sensitive marker of disability progression in ambulatory patients with multiple sclerosis. | EClinicalMedicine |