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

AIM: The purpose of this study is to evaluate the safety and pharmacokinetics of the novel morpholino oligomer NS‐089/NCNP‐02 which can induce exon 44 skipping, in patients with DMD. Additionally, we aimed to identify markers predictive of therapeutic efficacy and determine the optimal dosing for fu...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10275280/
https://www.ncbi.nlm.nih.gov/pubmed/37326950
http://dx.doi.org/10.1002/npr2.12335

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