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与PIK3CA相关的过度生长综合征患者的向治疗

Quitterie Venot1, Thomas Blanc1,2,3, Smail Hadj Rabia2,4,5

  • 1INSERM U1151, Institut Necker Enfants Malades, Paris, France.

Nature
|June 15, 2018
PubMed
概括

这是一种罕见的遗传疾病,可通过抑制PIK3CA有效治疗. 这种与PIK3CA相关的过度生长综合征 (PROS) 治疗显示患者的症状明显改善,器官功能障碍减少.

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