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
Muscle & Nerve（2021）
关键词
casimersen,Duchenne muscular dystrophy,exon skipping,phosphorodiamidate morpholino oligomer,RNA oligonucleotide
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