Lindsay N Alfano1, Linda P Lowes, Kevin M Flanigan, Jerry R Mendell. 1. Center for Gene Therapy and the Paul D. Wellstone Cooperative Research Center, The Research Institute at Nationwide Children's Hospital, 700 Children's Drive, Columbus, Ohio 43205, USA.
Abstract
INTRODUCTION: The correlation of strength with performance on functional outcomes has not been evaluated in Becker muscular dystrophy (BMD), yet the determination of proper outcome measures is critical to the success of upcoming trials designed to improve strength. METHODS: Lower extremity strength and performance on functional outcome measures were tested in 25 ambulatory subjects with BMD. RESULTS: All subjects demonstrated marked knee extensor and flexor muscle weakness. Knee extensor strength was correlated with performance on the Berg Balance Scale and stair climbing. Knee flexor strength was highly correlated with performance on all functional outcomes, including timed walking distances. CONCLUSIONS: This profile differs from that previously reported in other neuromuscular diseases and demonstrates the importance of designing outcome measures for clinical trials in muscle disorders with considerations for the disease process, the mode, and the target of intervention. The findings emphasize that 1 set of outcomes is not appropriate for all neuromuscular diseases.
INTRODUCTION: The correlation of strength with performance on functional outcomes has not been evaluated in Becker muscular dystrophy (BMD), yet the determination of proper outcome measures is critical to the success of upcoming trials designed to improve strength. METHODS: Lower extremity strength and performance on functional outcome measures were tested in 25 ambulatory subjects with BMD. RESULTS: All subjects demonstrated marked knee extensor and flexor muscle weakness. Knee extensor strength was correlated with performance on the Berg Balance Scale and stair climbing. Knee flexor strength was highly correlated with performance on all functional outcomes, including timed walking distances. CONCLUSIONS: This profile differs from that previously reported in other neuromuscular diseases and demonstrates the importance of designing outcome measures for clinical trials in muscle disorders with considerations for the disease process, the mode, and the target of intervention. The findings emphasize that 1 set of outcomes is not appropriate for all neuromuscular diseases.
Authors: Marein M Favejee; Jan C van der Meijden; Michelle E Kruijshaar; Dimitris Rizopoulos; Ans T van der Ploeg; Johannes B J Bussmann Journal: Phys Ther Date: 2018-11-01
Authors: Jessica W Witherspoon; Julie S Rekant; Paul G Wakim; Ruhi Vasavada; Melissa Waite; Irene Chrismer; Monique O Shelton; Minal S Jain; Katherine G Meilleur Journal: Front Neurol Date: 2019-12-10 Impact factor: 4.003