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

Abstract 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 dosin...

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Auteurs principaux:	Takami Ishizuka, Hirofumi Komaki, Yasuko Asahina, Harumasa Nakamura, Norio Motohashi, Eri Takeshita, Yuko Shimizu‐Motohashi, Akihiko Ishiyama, Chihiro Yonee, Shinsuke Maruyama, Eisuke Hida, Yoshitsugu Aoki
Format:	Artigo
Langue:	Inglês
Publié:	Wiley 2023-06-01
Collection:	Neuropsychopharmacology Reports
Sujets:	antisense oligonucleotide DMD Duchenne muscular dystrophy exon 44 exon‐skipping first‐in‐human study
Accès en ligne:	https://doi.org/10.1002/npr2.12335
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