Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial

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作者： Wagner, Kathryn R. ^[1] ; Kuntz, Nancy L. ^[2] ; Koenig, Erica ^[3] ; East, Lilly ^[4] ; Upadhyay, Sameer ^[5] ; Han, Baoguang ^[6] ; Shieh, Perry B. ^[7]

作者单位： Univ Calif Los Angeles, Dept Neurol, Los Angeles, CA 90024 USA ^[1] Ann & Robert H Lurie Childrens Hosp, Chicago, IL USA ^[2] Sarepta Therapeut Inc, Clin Dev, Cambridge, MA USA ^[3] Sarepta Therapeut Inc, Pharmacovigilance, Cambridge, MA USA ^[4] Kennedy Krieger Inst, Ctr Genet Muscle Disorders, Baltimore, MD USA ^[5] Sarepta Therapeut Inc, Clin Pharmacol, Cambridge, MA USA ^[6] Sarepta Therapeut Inc, Biostat, Cambridge, MA USA ^[7]

关键词 casimersen Duchenne muscular dystrophy exon skipping phosphorodiamidate morpholino oligomer RNA oligonucleotide

发布时间 2021-12-08

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