Background: Cerebral palsy is a life-long condition that causes heterogeneous motor disorders. Motor rehabilitation interventions must be adapted to the topography of the symptoms, ambulatory capacity, and age of the individual. Current guidelines do not differentiate between the different profiles of individuals with cerebral palsy, which limits their implementation.
View Article and Find Full Text PDFObjectives: Because X-linked myotubular myopathy (XLMTM) is a rare neuromuscular disease caused by mutations in the gene with a large phenotypic heterogeneity, to ensure clinical trial readiness, it was mandatory to better quantify disease burden and determine best outcome measures.
Methods: We designed an international prospective and longitudinal natural history study in patients with XLMTM and assessed muscle strength and motor and respiratory functions over the first year of follow-up. The humoral immunity against adeno-associated virus serotype 8 was also monitored.
Objective: To validate a useful version of the Motor Function Measure (MFM) in children with neuromuscular diseases aged <7 years old.
Design: Two prospective cohort studies that documented the MFM completion of children aged between 2 and 7 years old.
Setting: French-speaking rehabilitation departments from France, Belgium, and Switzerland.
Aims: Our objective was to clarify the clinical heterogeneity in Duchenne muscular dystrophy (DMD).
Methods: The French dystrophinopathy database provided clinical, histochemical and molecular data of 278 DMD patients (mean longitudinal follow-up: 14.2 years).
Aim: To assess changes in motor function in patients with Duchenne muscular dystrophy using the Motor Function Measure (MFM).
Method: Three studies were performed. Two studies included only physiotherapy-treated patients, with 13 patients (males mean age 11y 7mo, SD 1y 10mo, range 8-14y) in the 3-month study and 41 patients (males mean age 14y 1mo, SD 5y 5mo, range 6-32y) in the 1-year study.
Background: To examine feasibility and relevance of a multidimensional outcome assessment approach using instrumented 3-dimensional gait analysis, via the Gillette Gait Index (GGI), and a set of validated functional and health-related quality of life tools in diplegic cerebral palsy children, before introduction as a nationwide evaluation set.
Methods: A 3-year prospective government-funded multicenter study was conducted, recruiting patients during a 9-month period classified using the Gross Motor Function Classification System and the Rodda et al sagittal walking patterns. The Gross Motor Classification System Dimensions D and E, the 10-level Gillette Functional Assessment Questionnaire, the Energy Expenditure Index (EEI), the GGI out of 3D gait analysis, and health-related quality of life, assessed by self or proxy with the questionnaire "Vècu et Santè Perçu de l'Adolescent," were selected for the study.