Givosiran in acute intermittent porphyria: A personalized medicine approach

Antoine Poli,Caroline Schmitt,Boualem Moulouel,Arienne Mirmiran,Neila Talbi,Sophie Rivière, Diane Cerutti, Isabelle Bouchoule,Anthony Faivre,Vincent Grobost,Claire Douillard, Francis Duchêne,Valeria Fiorentino,Thierry Dupré,Hana Manceau,Katell Peoc'h, Hervé Puy,Thibaud Lefebvre, Laurent Gouya

Molecular Genetics and Metabolism（2022）

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摘要

•Givosiran sustained activity allows to reduce dosing frequency•Reduced dosing does not impair clinical or biological response to treatment•Givosiran is most effective when given early in the disease course•Givosiran is associated with severe hyperhomocysteinemia that responds to B6

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关键词

Acute intermittent porphyria,Givosiran,Homocysteine

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