Responsiveness of the Motor Function Measure in Patients With Spinal Muscular Atrophy
作者： Carole Vuillerot , Christine Payan , Jean Iwaz , René Ecochard , Carole Bérard
DOI: 10.1016/J.APMR.2013.01.014
关键词:
摘要: Objective: To assess the ability of Motor Function Measure (MFM) to detect changes in progression spinal muscular atrophy (SMA). Design: Observational, retrospective, multicenter cohort study. Setting: Seventeen departments pediatric physical medicine. Participants: Volunteer patients with SMA (NZ112) aged 5.7 59 years no treatment other than therapy and nutritional or respiratory assistance. Interventions: Not applicable. Main Outcome Measures: The distributions MFM scores (total score 3 subscores) were analyzed per subtype. relationships between age studied. slopes (reflecting responsiveness) estimated at least 6 months’ follow-up 2 MFMs. Hypothetical sample sizes for specific effect clinical trial scenarios are given. Results
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