Viltolarsen in Japanese Duchenne Muscular Dystrophy Patients: A Phase 1/2 Study.

Hirofumi Komaki,Yasuhiro Takeshima,Tsuyoshi Matsumura,Shiro Ozasa,Michinori Funato,Eri Takeshita,Yasuyuki Iwata,Hiroyuki Yajima, Yoichi Egawa, Takuya Toramoto, Masaya Tajima,Shinichi Takeda

Annals of clinical and translational neurology（2020）

引用 44|浏览24

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摘要

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). We investigated viltolarsen’s ability to induce dystrophin expression and examined its safety in DMD patients.

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