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

• मुख्य लेखकों: Takami Ishizuka (लेखक), Hirofumi Komaki (लेखक), Yasuko Asahina (लेखक), Harumasa Nakamura (लेखक), Norio Motohashi (लेखक), Eri Takeshita (लेखक), Yuko Shimizu‐Motohashi (लेखक), Akihiko Ishiyama (लेखक), Chihiro Yonee (लेखक), Shinsuke Maruyama (लेखक), Eisuke Hida (लेखक), Yoshitsugu Aoki (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Wiley, 2023-06-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...