Article
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.
Neuropsychopharmacology reports - 1 Jun 2023
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
Abstract excerpt
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 future studies. METHODS: This is an open-label, dose-escalation, two-center phase I/II trial in ambulant patients with...
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