Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study

Komaki H, Takeshima Y, Matsumura T, et al. Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study. Annals of clinical and translational neurology 2020   Abstract OBJECTIVE: The novel morpholino antisense oligonucleotide viltolarsen targets exon 53 of the dystrophin gene, and could be an effective treatment for patients with Duchenne muscular dystrophy (DMD). […]