Anne M Connolly, Elizabeth C Malkus, Jerry R Mendell, Kevin M Flanigan, J Philip Miller, Jeanine R Schierbecker, Catherine A Siener, Paul T Golumbek, Craig M Zaidman, Craig M Mcdonald, Linda Johnson, Alina Nicorici, Peter I Karachunski, John W Day, Jason M Kelecic, Linda P Lowes, Lindsay N Alfano, Basil T Darras, Peter B Kang, Janet Quigley, Amy E Pasternak, Julaine M Florence
INTRODUCTION: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. METHODS: Non-ambulatory boys/men with DMD (N = 91; 16.7 ± 4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured...
April 2015: Muscle & Nerve