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Updated: Sep 16, 2025

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SFX-01对由Shp2激活突变引起的骨髓增殖性疾病具有治疗作用

Hyun-Ju Cho1, Joy Smith1,2, Christopher H Switzer3

  • 1William Harvey Research Institute, Faculty of Medicine and Dentistry, Queen Mary University of London, London, UK.

EMBO molecular medicine
|July 10, 2025
PubMed
概括

SFX-01是一种新型治疗药物,可以抑制过度活跃的Shp2酸酶,该酶与Noonan综合征和青少年骨髓 mononcytic白血病 (JMML) 等儿童疾病有关. 这种化合物在临床前模型中使关键疾病标志物正常化,提供了潜在的新疗法.

关键词:
骨髓增殖性疾病 骨髓增殖性疾病诺南综合征是什么意思在SFX-01中,它是最重要的.一个shp2一个shp2硫甲是一种硫甲.

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