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Diabetes Metab Res Rev. 2019, 35:e3140. 10.1002\/dmrr.3140","journal-title":"Diabetes Metab Res Rev"},{"key":"ref53","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2021.10.005","article-title":"Linking liver metabolic and vascular disease via bile acid signaling","volume":"28","author":"Fiorucci S","year":"2022","unstructured":"Fiorucci S, Distrutti E. Linking liver metabolic and vascular disease via bile acid signaling. Trends Mol Med. 2022, 28:51-66. 10.1016\/j.molmed.2021.10.005","journal-title":"Trends Mol Med"},{"key":"ref54","doi-asserted-by":"publisher","DOI":"10.1097\/HC9.0000000000000213","article-title":"Hepatic farnesoid X receptor is necessary to facilitate ductular reaction and expression of heme biosynthetic genes","volume":"7","author":"Dean AE","year":"2023","unstructured":"Dean AE, Jungwirth E, Panzitt K, Wagner M, Anakk S. Hepatic farnesoid X receptor is necessary to facilitate ductular reaction and expression of heme biosynthetic genes. Hepatol Commun. 2023, 7:10. 10.1097\/HC9.0000000000000213","journal-title":"Hepatol Commun"},{"key":"ref55","doi-asserted-by":"publisher","DOI":"10.1002\/hep.21879","article-title":"Regulation of human liver delta-aminolevulinic acid synthase by bile acids","volume":"46","author":"Peyer AK","year":"2007","unstructured":"Peyer AK, Jung D, Beer M, et al.. Regulation of human liver delta-aminolevulinic acid synthase by bile acids. Hepatology. 2007, 46:1960-70. 10.1002\/hep.21879","journal-title":"Hepatology"},{"key":"ref56","doi-asserted-by":"publisher","DOI":"10.1016\/s0168-8278(86)80496-2","article-title":"Chenodeoxycholic acid therapy in erythrohepatic protoporphyria","volume":"3","author":"Van Hattum J","year":"1986","unstructured":"Van Hattum J, Baart de la Faille H, Van den Berg JW, Edixhoven-Bosdijk A, Wilson JH. Chenodeoxycholic acid therapy in erythrohepatic protoporphyria. J Hepatol. 1986, 3:407-12. 10.1016\/s0168-8278(86)80496-2","journal-title":"J Hepatol"},{"key":"ref57","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.2597","article-title":"Givosiran likely inhibits cytochrome P450 more substantially than reported","volume":"112","author":"Bins S","year":"2022","unstructured":"Bins S, Sardh E, Langendonk JG. Givosiran likely inhibits cytochrome P450 more substantially than reported. Clin Pharmacol Ther. 2022, 112:24. 10.1002\/cpt.2597","journal-title":"Clin Pharmacol Ther"},{"key":"ref58","unstructured":"Conundrum. Accessed. 21.8.2024: https:\/\/www.dictionary.com\/e\/word-of-the-day\/conundrum-2022-02-11\/."},{"key":"ref59","doi-asserted-by":"publisher","DOI":"10.1136\/bmjgast-2024-ICPP.2","article-title":"Patient demographics and clinical characteristics at enrollment in ELEVATE, an international registry of acute hepatic porphyria","volume":"11","author":"Sardh E","year":"2024","unstructured":"Sardh E, Cassiman D, Gouya L, et al.. 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