Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

May 15th, 2020|Categories: Alternative splicing, Clinical Development, Delivery, Immune Stimulation, Medicinal Chemistry, Pre-Clinical Research, Ribozyme, Toxicology|

Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol. 2020 May 26 Pubmed

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