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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Bibliografische gegevens
Gepubliceerd in:Ann Clin Transl Neurol
Hoofdauteurs: Komaki, Hirofumi, Takeshima, Yasuhiro, Matsumura, Tsuyoshi, Ozasa, Shiro, Funato, Michinori, Takeshita, Eri, Iwata, Yasuyuki, Yajima, Hiroyuki, Egawa, Yoichi, Toramoto, Takuya, Tajima, Masaya, Takeda, Shinichi
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: John Wiley and Sons Inc. 2020
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Research Articles
Online toegang: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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