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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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science