Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up
Muscle & Nerve2016Vol. 54(4), pp. 681–689
Citations Over TimeTop 10% of 2016 papers
Anne M. Connolly, Julaine Florence, Craig M. Zaidman, Paul T. Golumbek, Jerry R. Mendell, Kevin M. Flanigan, Peter Karachunski, John Day, Craig M. McDonald, Basil T. Darras, Peter B. Kang, Catherine Siener, Rebecca K. Gadeken, Pallavi Anand, Jeanine Schierbecker, Elizabeth C. Malkus, Linda Lowes, Lindsay N. Alfano, Linda B. Johnson, Alina Nicorici, Jason M. Kelecic, Janet Quigley, Amy Pasternak, J. Philip Miller
Abstract
Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681-689, 2016.
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