Givosiran in acute intermittent porphyria: A personalized medicine approach

Poli, A; Schmitt, C; Moulouel, B; Mirmiran, A; Talbi, N; Riviere, S; Cerutti, D; Bouchoule, I; Faivre, A; Grobost, V; Douillard, C; Duchene, F; Fiorentino, V; Dupre, T; Manceau, H; Peoc'h, K; Puy, H; Lefebvre, T; Gouya, L

Poli, A (通讯作者)，Louis Mourier Hosp, Ctr Francais Porphyries, 178 Rue Renouillers, F-92700 Colombes, France.

MOLECULAR GENETICS AND METABOLISM, 2022; 135 (3): 206

Abstract

Background: In patients with acute intermittent porphyria (AIP), induction of delta aminolevulinic acid synthase 1 (ALAS1) leads to haem precursor acc......

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