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Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial.
Ishizuka, Takami; Komaki, Hirofumi; Asahina, Yasuko; Nakamura, Harumasa; Motohashi, Norio; Takeshita, Eri; Shimizu-Motohashi, Yuko; Ishiyama, Akihiko; Yonee, Chihiro; Maruyama, Shinsuke; Hida, Eisuke; Aoki, Yoshitsugu.
Afiliação
  • Ishizuka T; Clinical Research and Education Promotion Division, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Komaki H; Clinical Research and Education Promotion Division, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Asahina Y; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Nakamura H; Clinical Research and Education Promotion Division, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Motohashi N; Clinical Research and Education Promotion Division, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Takeshita E; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Shimizu-Motohashi Y; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Ishiyama A; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Yonee C; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Maruyama S; Department of Pediatrics, Graduate School of Medical and Dental Sciences, Kagoshima University, Kagoshima City, Kagoshima, Japan.
  • Hida E; Department of Pediatrics, Graduate School of Medical and Dental Sciences, Kagoshima University, Kagoshima City, Kagoshima, Japan.
  • Aoki Y; Department of Biostatistics and Data Science, Graduate School of Medicine, Osaka University, Osaka, Japan.
Neuropsychopharmacol Rep ; 43(2): 277-286, 2023 Jun.
Article em En | MEDLINE | ID: mdl-37326950
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Guideline / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Guideline / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article