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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2020
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Materias: | |
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 |
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