Safety, tolerability, and efficacy of viltolarsen in boys with duchenne muscular dystrophy amenable to exon 53 skipping: A phase 2 randomized clinical trial
Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly, Amy D. Harper, Jean K. Mah, Edward C. Smith, Craig M. McDonald, Craig M. Zaidman, Lauren P. Morgenroth, Hironori Osaki, Youhei Satou, Taishi Yamashita, Eric P. Hoffman
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