DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study
Neurology2016Vol. 87(4), pp. 401–409
Citations Over TimeTop 10% of 2016 papers
Luca Bello, Lauren P. Morgenroth, Heather Gordish‐Dressman, Eric P. Hoffman, Craig M. McDonald, Sebahattin Çirak, CINRG investigators, Avital Cnaan, Mathula Thangarajh, Richard T. Abresch, Erik Henricson, Venkatarman Viswanathan, Laura McAdam, Jean K. Mah, M. Tulinius, Monique M. Ryan, Yoram Nevo, Alberto Dubrovsky, Paula R. Clemens, Anne M. Connolly, Jean Teasley, T. E. Bertoríni, Richard Webster, Hanna Kolski, K. Gorni, Timothy Lotze, Peter Karachunski, John B. Bodensteiner, James J. Carlo
Abstract
As exon 44 skipping-amenable DMD has a later LoA, mutation-specific randomization and selection of placebo groups are essential for the success of clinical trials.
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