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Molecular Genetics and Metabolism|January 21, 2022
Givosiran in acute intermittent porphyria: A personalized medicine approachAntoine Poli, Caroline Schmitt, Boualem Moulouel, et al.Human Mutation|February 11, 2011
Sideroblastic anemia: molecular analysis of the ALAS2 gene in a series of 29 probands and functional studies of 10 missense mutationsSarah Ducamp, Caroline Kannengiesser, Mohamed Touati, et al.Hepatology (Baltimore, Md.)|September 13, 2019
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent AttacksLaurent Gouya, Paolo Ventura, Manisha Balwani, et al.Pageof 2