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PIK3CA関連の過剰成長症候群の患者に対する標的治療

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

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

Nature
|June 15, 2018
PubMed
まとめ

CLOVES症候群は希少な遺伝疾患で,PIK3CA抑制で効果的に治療されています. このPIK3CA関連過剰成長症候群 (PROS) 治療は,患者の症状の改善と臓器機能不全の減少を示した.

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