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A Phase I/IIa Clinical Trial in Duchenne Muscular Dystrophy Using Systemically Delivered Morpholino Antisense Oligomer to Skip Exon 53 (SKIP-NMD)

Francesco Muntoni,Thomas Voit,Laurent Servais,Pierre-Yves Baudin,Volker Straub,Eugenio Mercuri, Joseph Bain,George Dickson,David Vissiere,Eric Dorveaux,Edward M. Kaye, P. Duda,William van't Hoff

HUMAN GENE THERAPY CLINICAL DEVELOPMENT（2015）

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