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
系统性应用反义寡核苷酸NS-089/NCNP-02治疗杜氏肌营养不良症患者,以跳跃外显子44:一项I/II期临床试验的研究方案
期刊:Neuropsychopharmacology Reports
影响因子:1.5
doi:10.1002/npr2.12335
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