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Disclosures: Alyssa Goldberg: Nothing to Disclose, William Hollenbach: Nothing to Disclose, Cara Mack: Nothing to Disclose, Beth Tamburini: Nothing to Disclose 2499 REQUIRED DOSE AND EFFECTS OF NTBC TREATMENT FOR HEREDITARY TYROSINEMIA TYPE 1: CONFIRMATIONS AND SURPRISES FROM A NOVEL, REPRESENTATIVE HUMAN MODEL Farzaneh Tamnanloo 1 Quang Toan Pham 1 Marie-Agns MCallum 1 Denis Cyr 2 Paula Waters 2 Emilie Beaulieu 1 Yannick Doyon 3 Ugur Halac 4 Claudia Raggi 1 Massimiliano Paganelli 4 , 1 Liver Tissue Engineering and Cell Therapy Laboratory, CHU Sainte-Justine, Montral, Canada, 2 Department of Biochemistry, Universit de Sherbrooke, Sherbrooke, Canada, 3 Department of Molecular Medicine, Universit Laval, Quebec, Canada, 4 Department of Pediatrics, Universit de Montral, Montral, Canada Background: Hereditary Tyrosinemia Type 1 (HT1) is a severe genetic liver disorder caused by a deficiency in fumarylacetoacetate hydrolase (FAH), a key enzyme of tyrosine metabolic pathway

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Priebe A, Tan L, Wahl H, Kueck A, He G, Kwok R et al