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

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

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Detalhes bibliográficos
Publicado no:Ann Clin Transl Neurol
Main Authors: Komaki, Hirofumi, Takeshima, Yasuhiro, Matsumura, Tsuyoshi, Ozasa, Shiro, Funato, Michinori, Takeshita, Eri, Iwata, Yasuyuki, Yajima, Hiroyuki, Egawa, Yoichi, Toramoto, Takuya, Tajima, Masaya, Takeda, Shinichi
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7732240/
https://ncbi.nlm.nih.gov/pubmed/33285037
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51235
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