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

• Main Authors: Takami Ishizuka (Author), Hirofumi Komaki (Author), Yasuko Asahina (Author), Harumasa Nakamura (Author), Norio Motohashi (Author), Eri Takeshita (Author), Yuko Shimizu‐Motohashi (Author), Akihiko Ishiyama (Author), Chihiro Yonee (Author), Shinsuke Maruyama (Author), Eisuke Hida (Author), Yoshitsugu Aoki (Author)
• Format: Book
• Published: Wiley, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...