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Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy.
Annals of Clinical and Translational Neurology ( IF 5.3 ) Pub Date : 2020-01-20 , DOI: 10.1002/acn3.50978
Hirofumi Komaki 1 , Yoshihiro Maegaki 2 , Tsuyoshi Matsumura 3 , Kazuhiro Shiraishi 4 , Hiroyuki Awano 5 , Akinori Nakamura 6 , Satoru Kinoshita 7 , Katsuhisa Ogata 8 , Keiko Ishigaki 9 , Shinji Saitoh 10 , Michinori Funato 11 , Satoshi Kuru 12 , Takahiro Nakayama 13 , Yasuyuki Iwata 14 , Hiroyuki Yajima 14 , Shin'ichi Takeda 15
Affiliation  

Duchenne muscular dystrophy (DMD) is a progressive muscular disease characterized by chronic cycles of inflammatory and necrotic processes. Prostaglandin D2 (PGD2) is produced by hematopoietic PGD synthase (HPGDS), which is pathologically implicated in muscle necrosis. This randomized, double‐blind, placebo‐controlled early phase 2 study (NCT02752048) aimed to assess the efficacy and safety of the novel selective HPGDS inhibitor, TAS‐205, with exploratory measures in male DMD patients aged ≥5 years.

中文翻译:

TAS-205 在杜氏肌营养不良症患者中的早期 2 期试验。

杜氏肌营养不良症 (DMD) 是一种进行性肌肉疾病,其特征是炎症和坏死过程的慢性循环。前列腺素 D 2 (PGD 2 ) 由造血 PGD 合酶 (HPGDS) 产生,在病理上与肌肉坏死有关。这项随机、双盲、安慰剂对照的早期 2 期研究 (NCT02752048) 旨在评估新型选择性 HPGDS 抑制剂 TAS-205 的疗效和安全性,并在 5 岁以上男性 DMD 患者中进行探索性措施。
更新日期:2020-01-20
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