Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints
Annals of Clinical and Translational Neurology2019Vol. 6(6), pp. 1033–1045
Citations Over TimeTop 13% of 2019 papers
Alexander P. Murphy, Jasper M. Morrow, Julia R. Dahlqvist, Tanya Stojkovic, Tracey Willis, Christopher D. J. Sinclair, Stephen Wastling, Tarek Yousry, Michael G. Hanna, M. James, Anna Mayhew, Michelle Eagle, Laurence Edward Lee, Jean‐Yves Hogrel, Pierre G. Carlier, John S. Thornton, John Vissing, Kieren G. Hollingsworth, Volker Straub
Abstract
These results further support fat fraction measurements as a primary outcome measure alongside functional assessments. The most appropriate individual muscles are the vastus lateralis, gracilis, sartorius, and gastrocnemii. Using composite groups of lower leg muscles, thigh muscles, or triceps surae, yielded high standardized response means (SRMs). Over 6 years, quantitative fat fraction assessment demonstrated higher SRM values than seen in functional tests suggesting greater responsiveness to disease progression.
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