Abstract
The 32-item Motor Function Measure (MFM32) is an assessment of motor function, and its measurement properties were established in a broad neuromuscular disease population. This study sought to investigate the reliability, validity, and ability to detect change of MFM32 in individuals with Type 2 and non-ambulant Type 3 spinal muscular atrophy (SMA). Data were used from the Phase 2 study assessing the efficacy and safety of olesoxime. A total of 110 individuals with Type 2 or 3 SMA were included in the analyses. Test-retest reliability (intraclass-correlation coefficient in global impression-defined stable individuals), internal consistency (Cronbach’s alpha), convergent validity (Spearman rank order correlations with other measures), known-groups validity (analysis of covariance comparing Hammersmith Functional Motor Scale -defined groups), and ability to detect change (analysis of covariance comparing global impression-defined groups) were calculated. Strong evidence of test-retest reliability (intraclass-correlation coefficient = 0.93–0.95), internal consistency (Cronbach’s alpha = 0.89), convergent validity (Hammersmith Functional Motor Scale: rho = 0.87; forced vital capacity: rho = 0.61), known-groups validity (all p<0.0001), and ability to detect change (all p<0.001) were demonstrated. These results provide evidence of the MFM32’s measurement properties, supporting its use in longitudinal research in individuals with Type 2 and non-ambulant Type 3 SMA.
Highlights
Spinal muscular atrophy (SMA) is a rare and severe progressive neuromuscular disease that causes muscle atrophy and disease-related complications which affect the whole body [1, 2]
SMA is caused by reduced levels of the survival of motor neuron (SMN) protein due to deletions and/or mutations of the SMN1 gene [3]
The study was approved by local institutional review boards and ethics committees including Federal Institute for Drugs and Medical Devices (BfArM; Germany), Rejestracji Produktow Leczniczych Wyrobow Medycznych i Produktow Biobojczych (Poland), Division R&D Federal Agency for Medicines and Health Products (FAMHP; Belgium), Central Committee on Research Involving Human Subjects (CCMO; Netherlands), Medicines and Healthcare Products Regulatory Agency (United Kingdom), The French Health Product Safety Agency (AFSSAPS; France), and the Ethics Committee for Medical Experimentation at the Ospedale Pediatrico Bambino Gesu (Italy)
Summary
Spinal muscular atrophy (SMA) is a rare and severe progressive neuromuscular disease that causes muscle atrophy and disease-related complications which affect the whole body [1, 2]. SMA is caused by reduced levels of the survival of motor neuron (SMN) protein due to deletions and/or mutations of the SMN1 gene [3]. Validity and reliability of MFM32 in Type 2 and non-ambulant Type 3 SMA. Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_ development/who_we_are_how_we_work/clinical_ trials/our_commitment_to_data_sharing)
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