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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7732240/
https://www.ncbi.nlm.nih.gov/pubmed/33285037
http://dx.doi.org/10.1002/acn3.51235

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7732240/
https://www.ncbi.nlm.nih.gov/pubmed/33285037
http://dx.doi.org/10.1002/acn3.51235

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